Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy.

Humans Adrenoleukodystrophy / therapy Genetic Therapy Genetic Vectors Hematologic Neoplasms / therapy Male Lentivirus / genetics ATP Binding Cassette Transporter, Subfamily D, Member 1 / genetics Female Adolescent Child Adult Antigens, CD34 Myelodysplastic Syndromes / therapy Young Adult

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:
10 Oct 2024
Historique:
medline: 9 10 2024
pubmed: 9 10 2024
entrez: 9 10 2024
Statut: ppublish

Résumé

Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced with lentiviral vector containing We performed integration-site analysis, genetic studies, flow cytometry, and morphologic studies in peripheral-blood and bone marrow samples from patients who received eli-cel therapy in two completed phase 2-3 studies (ALD-102 and ALD-104) and an ongoing follow-up study (LTF-304) involving the patients in both ALD-102 and ALD-104. Hematologic cancer developed in 7 of 67 patients after the receipt of eli-cel (1 of 32 patients in the ALD-102 study and 6 of 35 patients in the ALD-104 study): myelodysplastic syndrome (MDS) with unilineage dysplasia in 2 patients at 14 and 26 months; MDS with excess blasts in 3 patients at 28, 42, and 92 months; MDS in 1 patient at 36 months; and acute myeloid leukemia (AML) in 1 patient at 57 months. In the 6 patients with available data, predominant clones contained lentiviral vector insertions at multiple loci, including at either Hematologic cancer developed in a subgroup of patients who were treated with eli-cel; the cases are associated with clonal vector insertions within oncogenes and clonal evolution with acquisition of somatic genetic defects. (Funded by Bluebird Bio; ALD-102, ALD-104, and LTF-304 ClinicalTrials.gov numbers, NCT01896102, NCT03852498, and NCT02698579, respectively.).

Sections du résumé

BACKGROUND BACKGROUND
Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced with lentiviral vector containing
METHODS METHODS
We performed integration-site analysis, genetic studies, flow cytometry, and morphologic studies in peripheral-blood and bone marrow samples from patients who received eli-cel therapy in two completed phase 2-3 studies (ALD-102 and ALD-104) and an ongoing follow-up study (LTF-304) involving the patients in both ALD-102 and ALD-104.
RESULTS RESULTS
Hematologic cancer developed in 7 of 67 patients after the receipt of eli-cel (1 of 32 patients in the ALD-102 study and 6 of 35 patients in the ALD-104 study): myelodysplastic syndrome (MDS) with unilineage dysplasia in 2 patients at 14 and 26 months; MDS with excess blasts in 3 patients at 28, 42, and 92 months; MDS in 1 patient at 36 months; and acute myeloid leukemia (AML) in 1 patient at 57 months. In the 6 patients with available data, predominant clones contained lentiviral vector insertions at multiple loci, including at either
CONCLUSIONS CONCLUSIONS
Hematologic cancer developed in a subgroup of patients who were treated with eli-cel; the cases are associated with clonal vector insertions within oncogenes and clonal evolution with acquisition of somatic genetic defects. (Funded by Bluebird Bio; ALD-102, ALD-104, and LTF-304 ClinicalTrials.gov numbers, NCT01896102, NCT03852498, and NCT02698579, respectively.).

Identifiants

DOI: 10.1056/NEJMoa2405541 PMID: 39383458
pubmed: 39383458
doi: 10.1056/NEJMoa2405541
doi:

Substances chimiques

ATP Binding Cassette Transporter, Subfamily D, Member 1 0
ABCD1 protein, human 0
Antigens, CD34 0

Banques de données

ClinicalTrials.gov
['NCT01896102', 'NCT03852498', 'NCT02698579']

Types de publication

Journal Article Clinical Trial, Phase II Clinical Trial, Phase III

Langues

eng

Sous-ensembles de citation

IM

Pagination

1287-1301

Informations de copyright

Copyright © 2024 Massachusetts Medical Society.

Auteurs

Christine N Duncan (CN)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Jacob R Bledsoe (JR)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Bartosz Grzywacz (B)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Amy Beckman (A)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Melissa Bonner (M)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Florian S Eichler (FS)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Jörn-Sven Kühl (JS)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Marian H Harris (MH)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Sarah Slauson (S)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Richard A Colvin (RA)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Vinod K Prasad (VK)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Gerald F Downey (GF)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Francis J Pierciey (FJ)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Melissa A Kinney (MA)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Marianna Foos (M)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Ankit Lodaya (A)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Nicole Floro (N)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Geoffrey Parsons (G)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Andrew C Dietz (AC)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Ashish O Gupta (AO)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Paul J Orchard (PJ)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

Himal L Thakar (HL)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).

David A Williams (DA)

From Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (C.N.D., D.A.W.), the Department of Pathology, Boston Children's Hospital (J.R.B., M.H.H.), and Massachusetts General Hospital and Harvard Medical School (F.S.E.) - all in Boston; the Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center (B.G., A.B.), and the Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota (A.O.G., P.J.O.) - both in Minneapolis; Bluebird Bio, Somerville, MA (M.B., S.S., R.A.C., V.K.P., G.F.D., F.J.P., M.A.K., M.F., A.L., N.F., G.P., A.C.D., H.L.T.); the Department of Pediatric Oncology, Hematology and Hemostaseology, Leipzig University Hospital, Leipzig, Germany (J.-S.K.); and the Division of Pediatric Transplant and Cellular Therapy, Duke University School of Medicine, Durham, NC (V.K.P.).
ORCID: 0000-0001-7057-6863

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Hematologic Cancer after Gene Therapy for...
questionsmedicales.fr › Maladies endocriniennes › Maladies des surrénales › Insuffisance surrénale › Adrénoleucodystrophie › Hematologic Cancer after Gene Therapy for...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Hematologic Cancer after Gene Therapy for...
questionsmedicales.fr › Phénomènes génétiques › Structures génétiques › Vecteurs génétiques › Hematologic Cancer after Gene Therapy for...
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