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Complete Genetic Correction of iPS Cells From Duchenne Muscular Dystrophy

8 Dec 2009

Abstract excerpt

Human artificial chromosome (HAC) has several advantages as a gene therapy vector, including stable episomal maintenance that avoids insertional mutations and the ability to carry large gene inserts including the regulatory elements. Induced pluripotent stem (iPS) cells have great potential for gene therapy, as such cells can be generated from the individual's own tissues, and when reintroduced can contribute to...

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PMID: 19997091
DOI: 10.1038/mt.2009.274

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