Safety and Clinical Benefit of HSC Gene Therapy for Wiskott-Aldrich Syndrome

Press brief by Francesca Ferrua, MD, at the American Society of Hematology (ASH) 2015 annual meeting. Dr Ferrua provides an update on the safety and eficacy results of a phase I/II clinical trial with hematopoietic stem cell (HSC) gene therapy (GT) for Wiskott-Aldrich Syndrome (WAS). In summary, 7 WAS patients showed that GT is well tolerated and lead to a sustained clinical benefit. The high level of gene transfer obtained with LV-WAS resulted in robust engraftment of transduced HSC, even when combined with reduced intensity conditioning.