Applications of CRISPR/Cas9 in retinal degenerative diseases.

  title={Applications of CRISPR/Cas9 in retinal degenerative diseases.},
  author={Ying-Qian Peng and Luo-sheng Tang and Shigeo Yoshida and Ye-Di Zhou},
  journal={International journal of ophthalmology},
  volume={10 4},
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… CONTINUE READING