Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome
SHB Abina, HB Gaspar, J Blondeau, L Caccavelli… - Jama, 2015 - jamanetwork.com
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 outcomes and safety of autologous HSC gene therapy
in Wiskott-Aldrich syndrome. Design, Setting, and Participants Gene-corrected autologous
HSCs were infused in 7 consecutive patients with severe Wiskott-Aldrich syndrome lacking …
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 outcomes and safety of autologous HSC gene therapy
in Wiskott-Aldrich syndrome. Design, Setting, and Participants Gene-corrected autologous
HSCs were infused in 7 consecutive patients with severe Wiskott-Aldrich syndrome lacking …
