Wiskott–Aldrich Syndrome Treated With Stem-cell Gene Therapy

by Dr. Trupti Shirole on  December 9, 2010 at 5:44 PM Clinical Trials News
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Two patients with Wiskott-Aldrich syndrome have been successfully treated with a transfusion of autologous, genetically modified hematopoietic stem cells (HSC). The patients' clinical condition markedly improved after the treatment.
 Wiskott–Aldrich Syndrome Treated With Stem-cell Gene Therapy
Wiskott–Aldrich Syndrome Treated With Stem-cell Gene Therapy
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The Wiskott-Aldrich syndrome (WAS) is an X-linked recessive primary immunodeficiency disorder. It is associated with thrombocytopenia, eczema and autoimmunity.

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Following the gene therapy a sustained expression of WAS protein expression in HSC, lymphoid and myeloid cells and platelets was found. With resolution of hemorrhagic diathesis, eczema, autoimmunity and predisposition to severe infection certain functional corrections had to be made in the T and B cells, natural killer (NK) cells and monocytes . Vector integration that targeted multiple genes controlling growth and immunologic responses in a persistently polyclonal hematopoiesis was showed by comprehensive insertion-site analysis.




Source: Medindia
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darsheel Tuesday, December 21, 2010

my son is 2 years old and suffers from wiskott aldrich syndrom. he has eczema, bleeds a little now and then from the nose. can he be cued by the gene therapy?

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