Curr Gene Ther. 2015 Jan 26. [Epub ahead of print]
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Genetic Engineering Publications - GEG Tech top picks
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This review provides a state-of-the-art overview of preclinical studies for treatment of rare genetic diseases based on the SB transposase system for stable correction of the genetic defect. Diseases affecting the blood system, the connective tissue, the immune system, the metabolism, and the nervous system and their treatment utilizing the SB transposase system are discussed.
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