Current approaches in CRISPR-Cas9 mediated gene editing for biomedical and therapeutic applications

清脆的 基因组编辑 Cas9 锌指核酸酶 生物 转录激活物样效应核酸酶 计算生物学 基因 遗传增强 遗传学 生物信息学
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 Rawal,Suresh Ramakrishna,Vijai Singh
出处
期刊:Journal of Controlled Release [Elsevier]
卷期号:343: 703-723 被引量:24
标识
DOI:10.1016/j.jconrel.2022.02.005
摘要

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 crucial aspect for directing the reprogrammed cells toward clinical trials. The CRISPR-Cas9 system has been promising as a tool for correcting gene mutations in maladies including blood disorders and muscular degeneration as well as neurological, cardiovascular, renal, genetic, stem cell, and optical diseases. In this review, we highlight recent developments and utilization of the CRISPR-Cas9 system in correcting or generating gene mutations to create model organisms to develop deeper insights into diseases, rescue normal gene functionality, and curb the progression of a disease. Delivery of CRISPR-components being a pivotal aspect in proving its effectiveness, various proven delivery systems have also been briefly discussed.
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