作者
Gargi Bhattacharjee, Nisarg Gohil, Khushal Khambhati, Indra Mani, Rupesh Maurya, Janardhan Keshav Karapurkar, Jigresh Gohil, Dinh-Toi Chu, Hue Vu-Thi, Khalid J Alzahrani, Pau-Loke Show, Rakesh M Rawal, Suresh Ramakrishna, Vijai Singh
发表日期
2022/3/1
来源
Journal of Controlled Release
卷号
343
页码范围
703-723
出版商
Elsevier
简介
A single gene mutation can cause a number of human diseases that affect the quality of life. Until the development of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein (Cas) systems, it was challenging to correct a gene mutation to avoid a disease by reverting phenotypes. The advent of CRISPR technology has changed the field of gene editing, given its simplicity and intrinsic programmability, surpassing the limitations of both zinc-finger nuclease and transcription activator-like effector nuclease and becoming the method of choice for therapeutic gene editing by overcoming the bottlenecks of conventional gene-editing techniques. Currently, there is no commercially available medicinal cure to correct a gene mutation that corrects and reverses the abnormality of a gene’s function. Devising reprogramming strategies for faithful recapitulation of normal phenotypes is a …
引用总数
学术搜索中的文章