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

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

Hematology

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.

Please rate the quality of this content

As you found this content interesting...

Follow us on social media!

We are sorry that this content was not interesting for you!

Let us improve this content!

Tell us how we can improve this content?

Keep your finger on the pulse

Join Now

Elevating the Quality of Healthcare Globally

>