62. Hematopoietic Stem Cell Gene Therapy with Lentiviral Vector in 4 Patients with Cerebral X-Linked Adrenoleukodystrophy: Long-Term Outcome and Comparison of Efficacy with Allogeneic Hematopoietic Stem Cell Transplantation

Paul Orchard,1 Nathalie Cartier,2 Salima Hacein-Bey-Abina,3 Joanne Kurtzberg,4 Maria Escolar,5 Jakub Tolar,1 André Baruchel,6 Jean-Hughes Dalle,6 Gérard Michel,7 Stéphane Blanche,8 Cynthia Bartholomä,9 Manfred Schmidt,9 Pierre Bougnères,2,10 Christof van Kalle,9 Marina Cavazzana-Calvo,3 Alain Fischer,8 Patrick Aubourg.2,11