基因编辑用于角膜疾病管理。

Sudhanshu P Raikwar, Apoorva S Raikwar, Shyam S Chaurasia, Rajiv R Mohan
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引用次数: 4

摘要

基因编辑最近成为一项很有前途的技术,它可以在基因组中精确地设计基因修饰,从而长期缓解角膜疾病。分子生物学的最新进展导致了聚集规则间隔短回文重复序列(crispr)和crispr相关系统、锌指核酸酶和转录激活因子样效应核酸酶的发展,开创了高通量体外和体内基因组工程的新时代。基因组编辑可以成功地用于破译疾病病理生理基础的复杂分子机制,开发创新的下一代基因疗法、干细胞再生疗法,以及针对角膜和其他眼部疾病的个性化医疗。在这篇综述中,我们描述了基因组编辑领域的最新进展,当前的挑战,以及未来角膜疾病个性化基因医学发展的前景。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。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Gene editing for corneal disease management.

Gene editing for corneal disease management.

Gene editing for corneal disease management.

Gene editing for corneal disease management.

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 in vitro and in vivo 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.

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