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英国《自然·通讯》杂志发表一项遗传学重要研究成果,一种基因组编辑方法能够阻止小鼠视网膜退化,进而拯救失明小鼠。其所述方法利用了CRISPR/Cas9基因治疗系统,可适用于导致色素性视网膜炎(失明的主要原因)的各种潜在遗传缺陷。色素性视网膜炎无法医治、不易觉察,对患者眼睛造成极大危害而且具有遗传性。该病特征就是视网膜退化,但由于色素性视网膜炎可能由60多种基因的突变引起,因此,想要制定针对性疗法
A major genetics study published in the journal Nature Communications in the United Kingdom, a genome editing method that blocks mouse retinal degeneration and thus saves blind mice. The method utilizes the CRISPR / Cas9 gene therapy system and is applicable to a variety of potential genetic defects that cause retinitis pigmentosa, the leading cause of blindness. Pigmented retinitis can not be healed, difficult to detect, causing great harm to the eyes of patients and is hereditary. The disease is characterized by retinal degeneration, but due to retinitis pigmentosa may be caused by mutations in more than 60 genes, so you want to develop targeted therapies