Raikwar Sudhanshu P, Raikwar Apoorva S, Chaurasia Shyam S, Mohan Rajiv R
Department of Veterinary Medicine and Surgery, College of Veterinary Medicine, University of Missouri, Columbia, MO 65211, United States.
Department of Microbiology, Roy J. and Lucille A. Carver College of Medicine, University of Iowa, Iowa City, IA 52242, United States.
World J Transl Med. 2016 Apr 12;5(1):1-13. doi: 10.5528/wjtm.v5.i1.1.
Gene editing has recently emerged as a promising technology to engineer genetic modifications precisely in the genome to achieve long-term relief from corneal disorders. Recent advances in the molecular biology leading to the development of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPRs) and CRISPR-associated systems, zinc finger nucleases and transcription activator like effector nucleases have ushered in a new era for high throughput and genome engineering. Genome editing can be successfully used to decipher complex molecular mechanisms underlying disease pathophysiology, develop innovative next generation gene therapy, stem cell-based regenerative therapy, and personalized medicine for corneal and other ocular diseases. In this review we describe latest developments in the field of genome editing, current challenges, and future prospects for the development of personalized gene-based medicine for corneal diseases. The gene editing approach is expected to revolutionize current diagnostic and treatment practices for curing blindness.
基因编辑最近已成为一项很有前景的技术，可在基因组中精确地进行基因改造，以实现角膜疾病的长期缓解。分子生物学的最新进展导致了成簇规律间隔短回文重复序列（CRISPRs）及CRISPR相关系统、锌指核酸酶和转录激活因子样效应物核酸酶的发展，开创了高通量和基因组工程的新时代。基因组编辑可成功用于解读疾病病理生理学背后复杂的分子机制，开发创新的下一代基因疗法、基于干细胞的再生疗法以及针对角膜和其他眼部疾病的个性化药物。在本综述中，我们描述了基因组编辑领域的最新进展、当前挑战以及角膜疾病个性化基因药物开发的未来前景。基因编辑方法有望彻底改变目前治疗失明的诊断和治疗方法。
