Application of CRISPR/Cas9 genome editing technology in hereditary retinal diseases

Authors: Sun Xihao,  Tang shibo,  Chen Jiansu
DOI: 10.3760/cma.j.cn115989-20201120-00787
Published 2023-09-10
Cite as Chin J Exp Ophthalmol, 2023, 41(9): 925-930.

Abstract                            Download PDF】 【Read Full Text

Several mutant genes for inherited retinal diseases have been identified, but effective treatments are still lacking.The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) system can edit human genomic DNA by nonhomologous end joining or homology-directed repair, offering more possibilities for the treatment of hereditary retinal diseases.CRISPR/Cas9 not only can genetically correct patient-derived induced pluripotent stem cells (iPSCs) to observe their differentiation into retinal cells thereby, thereby exploring the pathogenesis of the disease and implementing cell therapy, but can also be delivered to the body via vectors and directly act on target cells to achieve in vivo gene editing.CRISPR/Cas9 gene editing technology in hereditary retinal diseases has been mainly used in retinitis pigmentosa, hereditary X-linked juvenile retinoschisis, and Leber congenital amaurosis 10, of which the in vitro application of CRISPR/Cas9 for Leber congenital amaurosis 10 has entered the clinical trial stage.In this paper, we reviewed the mechanism and key advances of CRISPR/Cas9 and provided an overview of gene editing in IRDs.

Key words:

Gene editing; CRISPR/Cas9; Inherited retinal diseases

Contributor Information

Sun Xihao

Aier School of Ophthalmology, Central South University, Aier Eye Institute Changsha, Chiangsha 410015, China

Tang shibo

Aier School of Ophthalmology, Central South University, Aier Eye Institute Changsha, Chiangsha 410015, China

Chen Jiansu

Aier School of Ophthalmology, Central South University, Aier Eye Institute Changsha, Chiangsha 410015, China

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