Applications of CRISPR/Cas9 in retinal degenerative diseases | Zendy

Yingqian Peng | Zendy; Luo-Sheng Tang | Zendy; Shigeo Yoshida | Zendy; Yedi Zhou | Zendy

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Applications of CRISPR/Cas9 in retinal degenerative diseases

Author(s) -

Yingqian Peng,

Luo-Sheng Tang,

Shigeo Yoshida,

Yedi Zhou

Publication year - 2017

Publication title -

international journal of ophthalmology

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 0.634

H-Index - 29

eISSN - 2227-4898

pISSN - 2222-3959

DOI - 10.18240/ijo.2017.04.23

Subject(s) - crispr , retinitis pigmentosa , genome editing , cas9 , medicine , induced pluripotent stem cell , genetic enhancement , retinal degeneration , retinal , computational biology , bioinformatics , gene , neuroscience , genetics , ophthalmology , biology , embryonic stem cell

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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