Francesco Morena

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

Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy

Vasco Meneghini, Giacomo Frati, Davide Sala et al. · Stem Cells Translational Medicine · 2016 · 84 citations · Full text

Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy

A Ricca, Nicole Rufo, Silvia Ungari et al. · Human Molecular Genetics · 2015 · 64 citations · Full text

Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy

Annalisa Lattanzi, Camilla Salvagno, Claudio Maderna et al. · Human Molecular Genetics · 2014 · 58 citations · Full text