Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.

Child, Preschool Female Follow-Up Studies Genetic Therapy Genetic Vectors Glycosaminoglycans / urine Hematopoietic Stem Cell Transplantation Humans Iduronidase / deficiency Infant Lentivirus Male Mucopolysaccharidosis I / metabolism Mutation Stem Cell Transplantation Transplantation, Autologous

Journal

The New England journal of medicine
ISSN: 1533-4406
Titre abrégé: N Engl J Med
Pays: United States
ID NLM: 0255562

Informations de publication

Date de publication:
18 11 2021
Historique:
entrez: 17 11 2021
pubmed: 18 11 2021
medline: 15 12 2021
Statut: ppublish

Résumé

Allogeneic hematopoietic stem-cell transplantation is the standard of care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this treatment is only partially curative and is associated with complications. We are conducting an ongoing study involving eight children with MPSIH. At enrollment, the children lacked a suitable allogeneic donor and had a Developmental Quotient or Intelligence Quotient score above 70 (i.e., none had moderate or severe cognitive impairment). The children received autologous hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with an α-L-iduronidase (IDUA)-encoding lentiviral vector after myeloablative conditioning. Safety and correction of blood IDUA activity up to supraphysiologic levels were the primary end points. Clearance of lysosomal storage material as well as skeletal and neurophysiological development were assessed as secondary and exploratory end points. The planned duration of the study is 5 years. We now report interim results. The children's mean (±SD) age at the time of HSPC gene therapy was 1.9±0.5 years. At a median follow-up of 2.10 years, the procedure had a safety profile similar to that known for autologous hematopoietic stem-cell transplantation. All the patients showed prompt and sustained engraftment of gene-corrected cells and had supraphysiologic blood IDUA activity within a month, which was maintained up to the latest follow-up. Urinary glycosaminoglycan (GAG) excretion decreased steeply, reaching normal levels at 12 months in four of five patients who could be evaluated. Previously undetectable levels of IDUA activity in the cerebrospinal fluid became detectable after gene therapy and were associated with local clearance of GAGs. Patients showed stable cognitive performance, stable motor skills corresponding to continued motor development, improved or stable findings on magnetic resonance imaging of the brain and spine, reduced joint stiffness, and normal growth in line with World Health Organization growth charts. The delivery of HSPC gene therapy in patients with MPSIH resulted in extensive metabolic correction in peripheral tissues and the central nervous system. (Funded by Fondazione Telethon and others; ClinicalTrials.gov number, NCT03488394; EudraCT number, 2017-002430-23.).

Sections du résumé

BACKGROUND
Allogeneic hematopoietic stem-cell transplantation is the standard of care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this treatment is only partially curative and is associated with complications.
METHODS
We are conducting an ongoing study involving eight children with MPSIH. At enrollment, the children lacked a suitable allogeneic donor and had a Developmental Quotient or Intelligence Quotient score above 70 (i.e., none had moderate or severe cognitive impairment). The children received autologous hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with an α-L-iduronidase (IDUA)-encoding lentiviral vector after myeloablative conditioning. Safety and correction of blood IDUA activity up to supraphysiologic levels were the primary end points. Clearance of lysosomal storage material as well as skeletal and neurophysiological development were assessed as secondary and exploratory end points. The planned duration of the study is 5 years.
RESULTS
We now report interim results. The children's mean (±SD) age at the time of HSPC gene therapy was 1.9±0.5 years. At a median follow-up of 2.10 years, the procedure had a safety profile similar to that known for autologous hematopoietic stem-cell transplantation. All the patients showed prompt and sustained engraftment of gene-corrected cells and had supraphysiologic blood IDUA activity within a month, which was maintained up to the latest follow-up. Urinary glycosaminoglycan (GAG) excretion decreased steeply, reaching normal levels at 12 months in four of five patients who could be evaluated. Previously undetectable levels of IDUA activity in the cerebrospinal fluid became detectable after gene therapy and were associated with local clearance of GAGs. Patients showed stable cognitive performance, stable motor skills corresponding to continued motor development, improved or stable findings on magnetic resonance imaging of the brain and spine, reduced joint stiffness, and normal growth in line with World Health Organization growth charts.
CONCLUSIONS
The delivery of HSPC gene therapy in patients with MPSIH resulted in extensive metabolic correction in peripheral tissues and the central nervous system. (Funded by Fondazione Telethon and others; ClinicalTrials.gov number, NCT03488394; EudraCT number, 2017-002430-23.).

Identifiants

DOI: 10.1056/NEJMoa2106596 PMID: 34788506
pubmed: 34788506
doi: 10.1056/NEJMoa2106596
doi:

Substances chimiques

Glycosaminoglycans 0
IDUA protein, human EC 3.2.1.76
Iduronidase EC 3.2.1.76

Banques de données

ClinicalTrials.gov
['NCT03488394']
EudraCT
['2017-002430-23']

Types de publication

Clinical Trial, Phase I Clinical Trial, Phase II Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1929-1940

Investigateurs

Federica Basilico (F)
Tatiana Bykova (T)
Maria Rosa Calvi (MR)
Martin Castelle (M)
Maria Della Bona (MD)
Narcisse Elenga (N)
Javier De Las Heras (J)
Silvia Funghini (S)
Urh Groselj (U)
Sabrina Malvagia (S)
Elise Martin (E)
Svetlana Mikhaylova (S)
Benedicte Neven (B)
Falucar Njuieyon (F)
Daniela Ombrone (D)
Marta Serafini (M)
Galit Tal (G)
Ilaria Visagalli (I)

Commentaires et corrections

Type : CommentIn

Informations de copyright

Copyright © 2021 Massachusetts Medical Society.

Auteurs

Bernhard Gentner (B)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
ORCID: 0000-0001-6024-4718

Francesca Tucci (F)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Stefania Galimberti (S)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Francesca Fumagalli (F)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Maurizio De Pellegrin (M)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Paolo Silvani (P)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Chiara Camesasca (C)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Silvia Pontesilli (S)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Silvia Darin (S)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Francesca Ciotti (F)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Marina Sarzana (M)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Giulia Consiglieri (G)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Chiara Filisetti (C)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Giulia Forni (G)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Laura Passerini (L)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Daniela Tomasoni (D)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Daniela Cesana (D)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Andrea Calabria (A)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Giulio Spinozzi (G)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Maria-Pia Cicalese (MP)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Valeria Calbi (V)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Maddalena Migliavacca (M)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Federica Barzaghi (F)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Francesca Ferrua (F)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Vera Gallo (V)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Simona Miglietta (S)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Erika Zonari (E)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Patali S Cheruku (PS)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Claudia Forni (C)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Marcella Facchini (M)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Ambra Corti (A)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Michela Gabaldo (M)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Stefano Zancan (S)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Serena Gasperini (S)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Attilio Rovelli (A)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Jaap-Jan Boelens (JJ)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.
ORCID: 0000-0003-2232-6952

Simon A Jones (SA)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Robert Wynn (R)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Cristina Baldoli (C)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Eugenio Montini (E)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Silvia Gregori (S)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Fabio Ciceri (F)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Maria G Valsecchi (MG)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Giancarlo la Marca (G)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Rossella Parini (R)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Luigi Naldini (L)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Alessandro Aiuti (A)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

Maria-Ester Bernardo (ME)

From the Departments of Hematology and Bone Marrow Transplantation (B.G., F. Ciceri), Pediatric Immunohematology and Bone Marrow Transplantation (F.T., F. Fumagalli, S.D., F. Ciotti, M.S., G.C., C. Filisetti, M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., A.A., M.-E.B.), Neurology and Neurophysiology (F. Fumagalli), Pediatric Orthopedics (M.D.P.), Anesthesia and Critical Care (P.S.), Pediatric Cardiology (C.C.), and Neuroradiology (S.P., C.B.) and the San Raffaele Telethon Institute for Gene Therapy (B.G., F.T., F. Fumagalli, L.P., D.T., D.C., A.C., G.S., M.-P.C., V.C., M.M., F.B., F. Ferrua, V.G., S.M., E.Z., P.S.C., M.F., A.C., E.M., S. Gregori, R.P., L.N., A.A., M.-E.B.), IRCCS San Raffaele Scientific Institute, Vita-Salute San Raffaele University (F. Ciceri, L.N., A.A., M.-E.B.), and Bicocca Bioinformatics, Biostatistics, and Bioimaging Center, School of Medicine and Surgery, University of Milano-Bicocca (S. Galimberti, M.G.V.), Milan, Fondazione Telethon, Rome (C. Forni, M.G., S.Z.), the Department of Experimental and Clinical Biomedical Sciences, University of Florence, and Newborn Screening, Biochemistry and Pharmacology Laboratory, Meyer Children's University Hospital (G.F., G.M.), Florence, and the Department of Pediatrics, Fondazione Monza e Brianza per il Bambino e la sua Mamma, San Gerardo Hospital, Monza (S. Gasperini, A.R., R.P.) - all in Italy; Stem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York (J.-J.B.); and the Willink Unit, Manchester Centre for Genomic Medicine, St. Mary's Hospital, Manchester University NHS Foundation Trust, University of Manchester (S.A.J.), and the Department of Blood and Marrow Transplantation, Royal Manchester Children's Hospital (R.W.) - both in Manchester, United Kingdom.

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Classifications MeSH

questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Enfant d'âge préscolaire › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études épidémiologiques › Études de cohortes › Études de suivi › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Phénomènes génétiques › Structures génétiques › Vecteurs génétiques › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Glucides › Polyosides › Glycosaminoglycanes › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation cellulaire › Transplantation de cellules souches › Transplantation de cellules souches hématopoïétiques › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Enzymes et coenzymes › Enzymes › Hydrolases › Glycosidases › L-iduronidase › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Personnes › Tranches d'âge › Nourrisson › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Virus › Virus à ARN › Retroviridae › Lentivirus › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Erreurs innées du métabolisme › Maladies lysosomiales › Mucopolysaccharidoses › Mucopolysaccharidose de type I › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Phénomènes génétiques › Variation génétique › Mutation › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation cellulaire › Transplantation de cellules souches › Hematopoietic Stem- and Progenitor-Cell Gene...
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation autologue › Hematopoietic Stem- and Progenitor-Cell Gene...