Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophy
Francesca Fumagalli, Valeria Calbi, Maria Sessa, Alberto A. Zambon, Elena Sophia Fratini, Maria Pia Cicalese, Vera Gallo, Francesca Ciotti, Marina Sarzana, Marcella Facchini, Sara Locatelli, Gigliola Antonioli, Stefano Zancan, Jesus Segovia, Laetitia Schwab, Gerald Downey, Fabio Ciceri, Massimo Filippi, Maria Grazia Natali Sora, Maria Ester Bernardo, Luigi Naldini, Alessandra Biffi, Alessandro Aiuti
Topics & Concepts
Metachromatic leukodystrophyGenetic enhancementProgenitor cellStem cellHematopoietic stem cellHaematopoiesisHematopoietic stem cell transplantationJuvenileMedicineCancer researchBiologyGenePathologyGeneticsRNA regulation and diseaseCytomegalovirus and herpesvirus researchNeutrophil, Myeloperoxidase and Oxidative Mechanisms