Sabrina Canale

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

C. von Kalle, G. Zanetti, E. Stupka, 2013, Science.

Alterations in the brain adenosine metabolism cause behavioral and neurological impairment in ADA-deficient mice and patients

L. Leocani, G. Comi, C. Baldoli, 2017, Scientific Reports.

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

P. Rancoita, L. Naldini, M. Roncarolo, 2016, The Lancet.

Metachromatic leukodystrophy – mutation analysis provides further evidence of genotype–phenotype correlation

M. Falautano, G. Comi, M. Roncarolo, 2008, Clinical genetics.

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

V. Gallo, P. Rancoita, C. Di Serio, 2022, The Lancet.

Metachromatic leukodystrophy: A single‐center longitudinal study of 45 patients

P. Rancoita, C. Di Serio, C. Baldoli, 2021, Journal of inherited metabolic disease.

Biffi Metachromatic Leukodystrophy Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits

Gianluigi Zanetti, Luca Biasco, Alessandro Aiuti, 2013 .