Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access-Reference-Cited by-同舟云学术

Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access

Published:2022-01 Issue:10322 Volume:399 Page:372-383
ISSN:0140-6736
Container-title:The Lancet
language:en
Short-container-title:The Lancet

Author:

Fumagalli Francesca,Calbi Valeria,Natali Sora Maria Grazia,Sessa Maria,Baldoli Cristina,Rancoita Paola Maria V,Ciotti Francesca,Sarzana Marina,Fraschini Maddalena,Zambon Alberto Andrea,Acquati Serena,Redaelli Daniela,Attanasio Vanessa,Miglietta Simona,De Mattia Fabiola,Barzaghi Federica,Ferrua Francesca,Migliavacca Maddalena,Tucci Francesca,Gallo Vera,Del Carro Ubaldo,Canale Sabrina,Spiga Ivana,Lorioli Laura,Recupero Salvatore,Fratini Elena Sophia,Morena Francesco,Silvani Paolo,Calvi Maria Rosa,Facchini Marcella,Locatelli Sara,Corti Ambra,Zancan Stefano,Antonioli Gigliola,Farinelli Giada,Gabaldo Michela,Garcia-Segovia Jesus,Schwab Laetitia C,Downey Gerald F,Filippi Massimo,Cicalese Maria Pia,Martino Sabata,Di Serio Clelia,Ciceri Fabio,Bernardo Maria Ester,Naldini Luigi,Biffi Alessandra,Aiuti Alessandro

Funder

GlaxoSmithKline Plc

Fondazione Telethon

Publisher

Elsevier BV

Subject

General Medicine

Reference34 articles.

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