Sabata Martino

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

Alessandra Biffi, Eugenio Montini, Laura Lorioli et al. · Science · 2013 · 1.2K citations · Full text

Cell Therapy, Immunology, Blood Cell +17

Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

Maria Sessa, Laura Lorioli, Francesca Fumagalli et al. · The Lancet · 2016 · 496 citations

Ad-hoc Analysis, Cell Therapy, Bone Marrow Failure +8

Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access

Francesca Fumagalli, Valeria Calbi, Maria Grazia Natali Sora et al. · The Lancet · 2022 · 266 citations · Full text

Identification of Hematopoietic Stem Cell–Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy

Bernhard Gentner, Ilaria Visigalli, Hidefumi Hiramatsu et al. · Science Translational Medicine · 2010 · 209 citations

In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

Antonella Consiglio, Angelo Quattrini, Sabata Martino et al. · Nature Medicine · 2001 · 202 citations · Full text

Vivo Gene Therapy, Experimental Neuropathology, Immunology +7