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Authors
Calabria, Andrea https://orcid.org/0000-0003-3515-3384
Spinozzi, Giulio https://orcid.org/0000-0002-4220-2474
Cesana, Daniela https://orcid.org/0000-0001-6366-4224
Buscaroli, Elena
Benedicenti, Fabrizio
Pais, Giulia https://orcid.org/0009-0005-5621-4803
Gazzo, Francesco https://orcid.org/0009-0000-4626-1386
Scala, Serena
Lidonnici, Maria Rosa https://orcid.org/0000-0003-3413-8414
Scaramuzza, Samantha
Albertini, Alessandra
Esposito, Simona https://orcid.org/0000-0003-1594-5835
Tucci, Francesca
Canarutto, Daniele
Omrani, Maryam
De Mattia, Fabiola
Dionisio, Francesca
Giannelli, Stefania
Marktel, Sarah
Fumagalli, Francesca https://orcid.org/0000-0001-7476-4087
Calbi, Valeria https://orcid.org/0000-0003-3514-666X
Cenciarelli, Sabina https://orcid.org/0000-0002-1413-4371
Ferrua, Francesca
Gentner, Bernhard https://orcid.org/0000-0001-6024-4718
Caravagna, Giulio https://orcid.org/0000-0003-4240-3265
Ciceri, Fabio https://orcid.org/0000-0003-0873-0123
Naldini, Luigi https://orcid.org/0000-0002-7835-527X
Ferrari, Giuliana
Aiuti, Alessandro https://orcid.org/0000-0002-5398-1717
Montini, Eugenio https://orcid.org/0000-0003-1771-6067
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Article History
Received: 16 September 2022
Accepted: 18 October 2024
First Online: 23 October 2024
Competing interests
: The San Raffaele Telethon Institute for GT (SR-Tiget) is a joint venture between the Telethon Foundation and Ospedale San Raffaele. Lentiviral vector-based gene therapy for metachromatic leukodystrophy (MLD), developed at SR-Tiget, was licensed to Orchard Therapeutics in 2018. Lentiviral vector-based gene therapy for Wiskott–Aldrich (WAS) syndrome was developed by Fondazione Telethon. Lentiviral vector-based gene therapy for β-thalassemia was developed at SR-Tiget. Gene therapy for MLD is approved in the EU (Libmeldy) and in the US (Lenmeldy). A.A. was the principal investigator of the pilot and pivotal SR-Tiget clinical trial of GT for MLD, WAS and β-Thal. The other authors declare no competing interests.
