Peng Ying-Qian, Tang Luo-Sheng, Yoshida Shigeo, Zhou Ye-Di
Department of Ophthalmology, the Second Xiangya Hospital, Central South University, Changsha 410011, Hunan Province, China.
Department of Ophthalmology, Kyushu University Graduate School of Medical Sciences, Fukuoka 812-8582, Japan.
Int J Ophthalmol. 2017 Apr 18;10(4):646-651. doi: 10.18240/ijo.2017.04.23. eCollection 2017.
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 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.
基因治疗是视网膜退行性疾病一种潜在的有效治疗方法。成簇规律间隔短回文重复序列（CRISPR）/CRISPR相关蛋白9（Cas9）系统已发展成为眼科研究中的一种新型基因组编辑工具。研究的最新进展表明，CRISPR/Cas9已应用于制备动物模型以及视网膜色素变性（RP）和莱伯先天性黑蒙（LCA）的基因治疗。通过与腺相关病毒（AAV）和诱导多能干细胞（iPSC）等其他技术相结合，它也被视为一种潜在的临床尝试。在这篇综述中，我们重点介绍了使用CRISPR/Cas9靶向视网膜变性的进一步前景要点。我们还强调了该技术在治疗视网膜退行性疾病中的潜在应用。
