G. Antonioli

Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy

A. Biffi, A. Aiuti, R. Miniero, 2019, Bone Marrow Transplantation.

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.

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.

Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy

G. Ardissino, L. Naldini, F. Morena, 2018, Bone Marrow Transplantation.

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

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

Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophy

M. Filippi, V. Gallo, L. Naldini, 2021 .