Applications of CRISPR/Cas9 in retinal degenerative diseases

Ying Qian Peng, Luo Sheng Tang, Shigeo Yoshida, Ye Di Zhou

研究成果: Contribution to journalArticle査読

12 被引用数 (Scopus)


• 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.

ジャーナルInternational Journal of Ophthalmology
出版ステータス出版済み - 4 18 2017

All Science Journal Classification (ASJC) codes

  • Ophthalmology

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