- PD-L1 genetic overexpression and pharmacological restoration in hematopoietic stem and progenitor cells cure autoimmune diabetes.
Sci Transl Med 2017;416:eaam7543,doi: 10.1126/scitranslmed.aam7543. - Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells.
Sci Adv 2017;3:e1701211. - Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2trial.
Lancet 2016; 388:476–487. - Autologous nonmyeloablative hematopoietic stem cell transplantation in new-onset type 1 diabetes: a multicenter analysis.
Diabetes 2014;63:3041-3046. - Therapeutic Benefit in Metachromatic Leukodystrophy Patients after Lentiviral Hematopoietic Stem Cell Gene Therapy.
Science 2013;341:1233158. - Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 2013;341:1233151.