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10.18240/ijo.2017.04.23

http://scihub22266oqcxt.onion/10.18240/ijo.2017.04.23
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C5406646!5406646!28503441
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suck abstract from ncbi


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pmid28503441      Int+J+Ophthalmol 2017 ; 10 (4): 646-51
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  • Applications of CRISPR/Cas9 in retinal degenerative diseases #MMPMID28503441
  • Peng YQ; Tang LS; Yoshida S; Zhou YD
  • Int J Ophthalmol 2017[]; 10 (4): 646-51 PMID28503441show ga
  • Gene therapy is a potentially effective treatment for retinal degenerative diseases. Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system has been developed as a new genome-editing tool in ophthalmic studies. Recent advances in researches showed that CRISPR/Cas9 has been applied in generating animal models as well as gene therapy in vivo of retinitis pigmentosa (RP) and leber congenital amaurosis (LCA). It has also been shown as a potential attempt for clinic by combining with other technologies such as adeno-associated virus (AAV) and induced pluripotent stem cells (iPSCs). In this review, we highlight the main points of further prospect of using CRISPR/Cas9 in targeting retinal degeneration. We also emphasize the potential applications of this technique in treating retinal degenerative diseases.
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