CRISPR/Cas9-mediated GJA8 knockout in rabbits recapitulates human congenital cataracts

• DOI: 10.1038/srep22024
• Publication Date: 2016-02-25T11:15:20Z
• Authors: Lin Yuan; Tingting Sui; Mao Chen; Jichao Deng; Yongye Huang; Jian Zeng; Qingyan Lv; Yuning Song; Zhanjun Li; Liangxue Lai
• Keywords: Gene knockout

Cataracts are the leading cause of vision loss in world, although surgical treatment can restore cataract patients. Until now, there have been no adequate animal models for vivo studies artificial lens safety and drug interactions. Genetic demonstrated that GJA8 is involved maintaining opacity proper development. In this study, a model with gene knockout was developed via co-injection Cas9/sgRNA mRNA into rabbit zygotes. Our results showed mutation efficiency locus reached 98.7% embryos 100% pups, demonstrating system highly efficient tool editing rabbits. agreement other studies, our genetic histology impaired function caused microphthalmia, small size cataracts. summary, novel cataracts will be an important drug-screening prevention treatment.