Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia.


Journal

Nature medicine
ISSN: 1546-170X
Titre abrégé: Nat Med
Pays: United States
ID NLM: 9502015

Informations de publication

Date de publication:
02 2019
Historique:
received: 25 07 2018
accepted: 13 11 2018
pubmed: 22 1 2019
medline: 11 5 2019
entrez: 22 1 2019
Statut: ppublish

Résumé

ß-thalassemia is caused by ß-globin gene mutations resulting in reduced (β

Identifiants

pubmed: 30664781
doi: 10.1038/s41591-018-0301-6
pii: 10.1038/s41591-018-0301-6
doi:

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

234-241

Auteurs

Sarah Marktel (S)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Haematology and BMT Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Samantha Scaramuzza (S)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Maria Pia Cicalese (MP)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Pediatric Immunohematology, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Fabio Giglio (F)

Haematology and BMT Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Stefania Galimberti (S)

Center of Biostatistics for Clinical Epidemiology, School of Medicine and Surgery, University of Milan-Bicocca, Milan, Italy.

Maria Rosa Lidonnici (MR)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Valeria Calbi (V)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Pediatric Immunohematology, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Andrea Assanelli (A)

Haematology and BMT Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Maria Ester Bernardo (ME)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Pediatric Immunohematology, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Claudia Rossi (C)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Andrea Calabria (A)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Raffaella Milani (R)

Blood Transfusion Service, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Salvatore Gattillo (S)

Blood Transfusion Service, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Fabrizio Benedicenti (F)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Giulio Spinozzi (G)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Annamaria Aprile (A)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Alessandra Bergami (A)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Miriam Casiraghi (M)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Pediatric Immunohematology, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Giulia Consiglieri (G)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Pediatric Immunohematology, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Nicoletta Masera (N)

Pediatric Department University of Milano-Bicocca, San Gerardo Hospital, Monza, Italy.

Emanuela D'Angelo (E)

Pediatric Clinic/DH, Fondazione IRCCS Ca' Granda, Milan, Italy.

Nadia Mirra (N)

Pediatric Clinic/DH, Fondazione IRCCS Ca' Granda, Milan, Italy.

Raffaella Origa (R)

Ospedale Pediatrico Microcitemico "A.Cao", A.O. "G.Brotzu", Cagliari, Italy.

Immacolata Tartaglione (I)

Department of Woman, Child and General and Specialist Surgery, Università degli studi della Campania "Luigi Vanvitelli", Napoli, Italy.

Silverio Perrotta (S)

Department of Woman, Child and General and Specialist Surgery, Università degli studi della Campania "Luigi Vanvitelli", Napoli, Italy.

Robert Winter (R)

Department of Chemical Pathology, Great Ormond Street Hospital NHS Foundation Trust, London, UK.

Milena Coppola (M)

Blood Transfusion Service, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Gianluca Viarengo (G)

Immunohematology and Transfusion Medicine Service, Fondazione IRCCS Policlinico S. Matteo, Pavia, Italy.

Luca Santoleri (L)

Blood Transfusion Service, IRCCS San Raffaele Scientific Institute, Milan, Italy.

Giovanna Graziadei (G)

Rare Disease Center, Fondazione IRCCS Ca' Granda, University of Milan, Milan, Italy.

Michela Gabaldo (M)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Maria Grazia Valsecchi (MG)

Center of Biostatistics for Clinical Epidemiology, School of Medicine and Surgery, University of Milan-Bicocca, Milan, Italy.

Eugenio Montini (E)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.

Luigi Naldini (L)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Vita-Salute San Raffaele University, Milan, Italy.

Maria Domenica Cappellini (MD)

Rare Disease Center, Fondazione IRCCS Ca' Granda, University of Milan, Milan, Italy.

Fabio Ciceri (F)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Haematology and BMT Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Vita-Salute San Raffaele University, Milan, Italy.

Alessandro Aiuti (A)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.
Pediatric Immunohematology, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Vita-Salute San Raffaele University, Milan, Italy.

Giuliana Ferrari (G)

San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy. ferrari.giuliana@hsr.it.
Vita-Salute San Raffaele University, Milan, Italy. ferrari.giuliana@hsr.it.

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