Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

Hacein-Bey Abina, Salima; Bobby Gaspar, H.; Blondeau, Johanna; Caccavelli, Laure; Charrier, Sabine; Buckland, Karen; Pïcard, Capucine; Six, Emmanuelle; Himoudi, Nourredine; Gilmour, Kimberly; McNicol, Anne‐Marie; Hara, Havinder; Xu‐Bayford, Jinhua; Rivat, Christine; Touzot, Fabien; Mavilio, Fulvio; Lim, Annick; Tréluyer, Jean‐Marc; Héritier, Sébastien; Lefrère, François; Magalon, Jérémy; Pengue-Koyi, Isabelle; Honnet, Géraldine; Blanche, Stéphane; A. Sherman, Eric; Male, Frances; C. Berry, Charles; Malani, Nirav; D. Bushman, Frederic; Fischer, Alain; J. Thrasher, Adrian; Galy, Anne; Cavazzana, Marina

doi:10.1001/jama.2015.3253

Public

Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

Published in JAMA • Apr 21, 2015

Authors:

Salima Hacein-Bey Abina

H. Bobby Gaspar

Johanna Blondeau

Abstract

IMPORTANCE: Wiskott-Aldrich syndrome is a rare primary immunodeficiency associated with severe microthrombocytopenia. Partially HLA antigen-matched allogeneic hematopoietic stem cell (HSC) transplantation is often curative but is associated with significant comorbidity. OBJECTIVE: To assess the outc...

Subject

Medicine

Wiskott–Aldrich syndrome

Genetic enhancement

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