Early sulfatide storage and progressive neuronal and glial cell dysfunction revealed by human iPSC-based models of metachromatic leukodystrophy are rescued by lentiviral-mediated restoration of ARSA activity

Early sulfatide storage and progressive neuronal and glial cell dysfunction revealed by human iPSC-based models of metachromatic leukodystrophy are rescued by lentiviral-mediated restoration of ARSA activity / Frati, ; G, ; Luciani, ; M, ; Meneghini, ; V, ; De, Cicco; S, ; Stahlman, ; M, ; Grossi, ; S, ; Morena, ; F, ; Blomqvist, ; M, ; Filocamo, ; M, ; Martino, ; S, ; Gritti, A. - In: HUMAN GENE THERAPY. - ISSN 1043-0342. - (2017).