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Genome-editing Technologies for Gene and Cell Therapy

12 Jan 2016

Abstract excerpt

Gene therapy has historically been defined as the addition of new genes to human cells. However, the recent advent of genome-editing technologies has enabled a new paradigm in which the sequence of the human genome can be precisely manipulated to achieve a therapeutic effect. This includes the correction of mutations that cause disease, the addition of therapeutic genes to specific sites in the genome, and the...

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PMID: 26755333
DOI: 10.1038/mt.2016.10

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