1040. Towards Gene Correction Therapy for Wiskott-Aldrich Syndrome Using Designed Zinc Finger Endonucleases

Wiskott-Aldrich Syndrome (WAS) is a severe X-linked primary immunodeficiency resulting from mutations in a hematopoietic- specific and developmentally-regulated gene. WAS is a prime candidate for treatment by autologous hematopoietic stem cell (HSC) gene editing since this disorder is caused by a single gene defect and corrected cells should have a strong selective advantage in vivo, especially at the level of T lymphocyte precursors and perhaps even at the level of HSCs.