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10.1038/nbt.2884

http://scihub22266oqcxt.onion/10.1038/nbt.2884
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C4157757!4157757!24681508
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suck abstract from ncbi


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pmid24681508      Nat+Biotechnol 2014 ; 32 (6): 551-3
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  • Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype #MMPMID24681508
  • Yin H; Xue W; Chen S; Bogorad RL; Benedetti E; Grompe M; Koteliansky V; Sharp PA; Jacks T; Anderson DG
  • Nat Biotechnol 2014[Jun]; 32 (6): 551-3 PMID24681508show ga
  • We demonstrate CRISPR-Cas9?mediated correction of a Fah mutation in hepatocytes in a mouse model of the human disease hereditary tyrosinemia. Delivery of components of the CRISPR-Cas9 system by hydrodynamic injection resulted in initial expression of the wild-type Fah protein in ~1/250 liver cells. Expansion of Fah-positive hepatocytes rescued the body weight loss phenotype. Our study indicates that CRISPR-Cas9?mediated genome editing is possible in adult animals and has potential for correction of human genetic diseases.
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