341. Lentiviral Vector-Mediated Gene Therapy as Treatment for Wiskott-Aldrich Syndrome (WAS): Pre-Clinical Studies in Human Cell Lines and WASp -/- Mice

Stephanie Humblet-Baron ,S Anover ,Kevin R Kipp ,Socheath Khim ,Alexander Astrakhan ,Dieter Kohn ,Terry B Strom ,Yatin M Vyas ,Wei Zhang ,Hermann R Ochs ,C Miao ,Peide D Ye ,Yulia Ovechkina ,Q M Zhu ,Fabio Candotti ,D Rawlings

doi:10.1016/j.ymthe.2005.06.344

341. Lentiviral Vector-Mediated Gene Therapy as Treatment for Wiskott-Aldrich Syndrome (WAS): Pre-Clinical Studies in Human Cell Lines and WASp -/- Mice

Stephanie Humblet-Baron , S Anover + Show 14 more

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https://doi.org/10.1016/j.ymthe.2005.06.344

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Journal: Molecular Therapy	Publication Date: May 1, 2005
License type: cc-by-nc-nd

#Wiskott-Aldrich Syndrome #Wiskott-Aldrich Syndrome Protein + Show 8 more

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Abstract

WAS is an immunodeficiency characterized by recurrent infection, thrombocytopenia, eczema, and increased susceptibility to autoimmunity and malignancy. WAS results from deficient function of the WAS protein (WASp) which functions in receptor-mediated actin cytoskeletal rearrangement. Gene therapy represents an attractive potential therapeutic approach for WAS patients.

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