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- W1999304857 abstract "Wiskott-Aldrich Syndrome (WAS) is an X-linked, recessive immune disorder caused by hypomorphic mutations in the gene encoding Wiskott-Aldrich Syndrome Protein (WASP), a major cytoskeletal regulator expressed in hematopoietic cells. WAS is therefore a good candidate for ex vivo gene correction therapy as the restoration of a single wild-type allele in a sub-population of patient cells is expected to provide therapeutic benefit (Charrier S et al., Gene Therapy Dec 2004).While gene replacement strategies seek to provide a new artificial copy of the defective gene, the gene correction approach aims to |[ldquo]|repair|[rdquo]| the clinically relevant mutations in the endogenous copy of the WASP gene, and thus restore normal gene function. This strategy eliminates the need for integrated transgenes, maintains the normal regulation of the corrected gene, and yet results in a permanent effect on cell phenotype." @default.
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- W1999304857 date "2005-05-01" @default.
- W1999304857 modified "2023-09-25" @default.
- W1999304857 title "337. Towards Gene Correction Therapy for Wiskott-Aldrich Syndrome with Engineered Zinc Finger Nucleases" @default.
- W1999304857 doi "https://doi.org/10.1016/j.ymthe.2005.06.340" @default.
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