Publications

  1. 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.
  2. Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells.
    Sci Adv 2017;3:e1701211.
  3. 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.
  4. Autologous nonmyeloablative hematopoietic stem cell transplantation in new-onset type 1 diabetes: a multicenter analysis.
    Diabetes 2014;63:3041-3046.
  5. Therapeutic Benefit in Metachromatic Leukodystrophy Patients after Lentiviral Hematopoietic Stem Cell Gene Therapy.
    Science 2013;341:1233158.
  6. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 2013;341:1233151.
  1. 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.
  2. Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells.
    Sci Adv 2017;3:e1701211.
  3. 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.
  4. Autologous nonmyeloablative hematopoietic stem cell transplantation in new-onset type 1 diabetes: a multicenter analysis.
    Diabetes 2014;63:3041-3046.
  5. Therapeutic Benefit in Metachromatic Leukodystrophy Patients after Lentiviral Hematopoietic Stem Cell Gene Therapy.
    Science 2013;341:1233158.
  6. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 2013;341:1233151.