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CRISPR-Cas9 在传染病和遗传疾病中的应用研究

CRISPR-Cas9 Probing of Infectious Diseases and Genetic Disorders.

机构信息

CSIR Institute for Genomics and Integrative Biology (IGIB), Mathura Road, Sukhdev Vihar, New Delhi, India.

Center for Stem Cell Research (CSCR), A Unit of inStem Bengaluru, Christian Medical College Campus, Vellore, Tamil Nadu, India.

出版信息

Indian J Pediatr. 2019 Dec;86(12):1131-1135. doi: 10.1007/s12098-019-03037-9. Epub 2019 Jul 31.

Abstract

The ability to precisely change the deoxyribonucleic acid (DNA) bases at specific sites offers tremendous advantages in the field of molecular biology and medical biotechnology. Identification of Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR), revelation of its role in prokaryotic adaptive immunity and subsequent conversion into genome and epigenome engineering system are the landmark research progresses of the decade. The possibilities of deciphering the molecular mechanisms of the disease, identifying the disease targets, generating the disease models, validating the drug targets, developing resistance to the infection and correcting the genotype have brought off much enthusiasm in the field of infectious diseases and genetic disorders. This review focuses on CRISPR/Cas9's impact in the field of infection and genetic disorders.

摘要

精确改变脱氧核糖核酸(DNA)碱基特定位置的能力在分子生物学和医学生物技术领域具有巨大优势。CRISPR(Clustered Regularly-Interspaced Short Palindromic Repeats)的鉴定,揭示了其在原核适应性免疫中的作用,以及随后转化为基因组和表观基因组工程系统,是这十年来的标志性研究进展。破译疾病的分子机制、鉴定疾病靶点、生成疾病模型、验证药物靶点、抗感染耐药和纠正基因型的可能性,在传染病和遗传疾病领域引发了极大的热情。本文重点介绍 CRISPR/Cas9 在感染和遗传疾病领域的影响。

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