Gene Therapy

Study Findings from K. Boztug et al Broaden Understanding of Gene Therapy

Published in Gene Therapy Weekly, January 20th, 2011

2011 JAN 20 -- According to recent research published in the New U.K. Journal of Medicine, "The Wiskott-Aldrich syndrome (WAS) is an X-linked recessive primary immunodeficiency disorder associated with thrombocytopenia, eczema, and autoimmunity. We treated two patients who had this disorder with a transfusion of autologous, genetically modified hematopoietic stem cells (HSC)."

"We found sustained expression of WAS protein expression in HSC, lymphoid and myeloid cells, and platelets after gene therapy. T and B cells, natural killer (NK) cells, and monocytes were functionally corrected. After treatment, the patients' clinical condition markedly...

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Source: Gene Therapy Weekly (2011-01-20)

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