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10.1038/nm.3793

http://scihub22266oqcxt.onion/10.1038/nm.3793
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C4492683!4492683!25654603
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suck abstract from ncbi


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pmid25654603      Nat+Med 2015 ; 21 (2): 121-31
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  • Therapeutic Genome Editing: Prospects and Challenges #MMPMID25654603
  • Cox DBT; Platt RJ; Zhang F
  • Nat Med 2015[Feb]; 21 (2): 121-31 PMID25654603show ga
  • Recent advances in the development of genome editing technologies based on programmable nucleases have significantly improved our ability to make precise changes in the genomes of eukaryotic cells. Genome editing is already broadening our ability to elucidate the contribution of genetics to disease by facilitating the creation of more accurate cellular and animal models of pathological processes. A particularly tantalizing application of programmable nucleases is the potential to directly correct genetic mutations in affected tissues and cells to treat diseases that are refractory to traditional therapies. Here we discuss current progress towards developing programmable nuclease-based therapies as well as future prospects and challenges.
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