Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in 5 patients treated under nominal compassionate use

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Bibliographic Details
Published in:Molecular genetics and metabolism 2023-02, Vol.138 (2), p.107044, Article 107044
Main Authors: Calbi, Valeria, Fumagalli, Francesca, Gallo, Vera, Recupero, Salvatore, De Mattia, Fabiola, Fratini, Elena Sophia, Corti, Ambra, Tarantola, Giulia, Sarzana, Marina, Ippolito, Alessia, Scarparo, Stefano, Ciotti, Francesca, Fraschini, Maddalena, Levi, Margherita, Sangalli, Mara, Morena, Francesco, Martino, Sabata, Sevin, Caroline, Narayanan, Vinodh, Adam, Etai, Emrick, Lisa, Giugliani, Roberto, Aiuti, Alessandro
Format: Article
Language:English
Online Access:Get full text
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ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2022.107044