Preliminary Communication
Outcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome
Salima Hacein-Bey Abina, PharmD, PhD; H. Bobby Gaspar, MRCP, PhD; Johanna Blondeau, MS; Laure Caccavelli, PhD; Sabine Charrier, PhD; Karen Buckland, PhD; Capucine Picard, MD, PhD; Emmanuelle Six, PhD; Nourredine Himoudi, PhD; Kimberly Gilmour, PhD; Anne-Marie McNicol, PhD; Havinder Hara, MS; Jinhua Xu-Bayford, DipHE; Christine Rivat, PhD; Fabien Touzot, MD, PhD; Fulvio Mavilio, PhD; Annick Lim, MS; Jean-Marc Treluyer, MD, PhD; Sébastien Héritier, MD; Francois Lefrère, MD; Jeremy Magalon, PharmD; Isabelle Pengue-Koyi, PharmD, PhD; Géraldine Honnet, MD; Stéphane Blanche, MD; Eric A. Sherman, BA; Frances Male, BA; Charles Berry, PhD; Nirav Malani, MS; Frederic D. Bushman, PhD; Alain Fischer, MD, PhD; Adrian J. Thrasher, MB, BS, PhD; Anne Galy, PhD; Marina Cavazzana, MD, PhD