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Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophy

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Bibliographic Details
Published in:	Molecular genetics and metabolism 2021-02, Vol.132 (2), p.S40-S40
Main Authors:	Fumagalli, Francesca, Calbi, Valeria, Sessa, Maria, Zambon, Alberto, Fratini, Elena, Cicalese, Maria Pia, Gallo, Vera, Ciotti, Francesca, Sarzana, Marina, Facchini, Marcella, Locatelli, Sara, Antonioli, Gigliola, Zancan, Stefano, Segovia, Jesus, Schwab, Laetitia C., Downey, Gerald, Ciceri, Fabio, Filippi, Massimo, Sora, Maria Grazia Natali, Bernardo, Maria Ester, Naldini, Luigi, Biffi, Alessandra, Aiuti, Alessandro
Format:	Article
Language:	English
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Online Access:	Get full text
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