Genetics in Medicine Open (Jan 2024)
P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up*
- Francesca Fumagalli,
- Valeria Calbi,
- Fabiola De Mattia,
- Alberto Zambon,
- Vera Gallo,
- Salvatore Recupero,
- Elena Sophia Fratini,
- Alessia Ippolito,
- Francesca Ciotti,
- Maddalena Fraschini,
- Marina Sarzana,
- Stefano Scarparo,
- Eugenio Montini,
- Sara Locatelli,
- Marcella Facchini,
- Alessandra Clerici,
- Francesco Morena,
- Sabata Martino,
- Muska Yarzi,
- Sean Moro,
- Andrew Shenker,
- Jean Brooks,
- Alan Richardson,
- Leslie Meltzer,
- Laura Campbell,
- Alessandro Aiuti
Affiliations
- Francesca Fumagalli
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Valeria Calbi
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Fabiola De Mattia
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Alberto Zambon
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Vera Gallo
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Salvatore Recupero
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Elena Sophia Fratini
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Alessia Ippolito
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Francesca Ciotti
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Maddalena Fraschini
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Marina Sarzana
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Stefano Scarparo
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Eugenio Montini
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Sara Locatelli
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Marcella Facchini
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Alessandra Clerici
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Francesco Morena
- University of Perugia, Perugia, Italy
- Sabata Martino
- University of Perugia, Perugia, Italy
- Muska Yarzi
- Orchard Therapeutics, London, England, UK
- Sean Moro
- Orchard Therapeutics, Boston, MA
- Andrew Shenker
- Clinical Consultant, Pennington, NJ
- Jean Brooks
- Orchard Therapeutics, London, England, UK
- Alan Richardson
- Orchard Therapeutics, London, England, UK
- Leslie Meltzer
- Orchard Therapeutics, Boston, MA
- Laura Campbell
- Orchard Therapeutics, London, England, UK
- Alessandro Aiuti
- San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, Italy
- Journal volume & issue
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Vol. 2
p. 100882
