Gene editing prospects for treating inherited retinal diseases

Retinal diseases can result from genetic mutations or combined risk factors, all causing vision impairment progressing to blindness. To date, available therapeutic approaches result in inefficient and expensive treatments. The CRISPR-Cas9 system, an RNA-guided genome editing technology, may be applied to precisely correct or remove disease-related genetic mutations, or induce permanent inhibition of factors promoting retinal degeneration. In this review, we will describe the CRISPR/Cas mechanism, as well as delivery tools. Also, we will focus on applications of CRISPR/Cas genome editing in the retina and in patient-derived induced pluripotent stem cells, highlighting the potential and future challenges of this technology to tackle retinal diseases. (By Dr. Alessandra Recchia, https://jmg.bmj.com/content/early/2019/12/18/jmedgenet-2019-106473 )

 

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