CRISPR/Cas9 System and its Research Progress in Gene Therapy

Author(s): Wenlou Liu, Chunsheng Yang, Yanqun Liu, Guan Jiang*.

Journal Name: Anti-Cancer Agents in Medicinal Chemistry
(Formerly Current Medicinal Chemistry - Anti-Cancer Agents)

Volume 19 , Issue 16 , 2019

Become EABM
Become Reviewer

Graphical Abstract:


Abstract:

Genome editing refers to changing the genome sequence of an organism by knockout, insertion, and site mutation, resulting in changes in the genetic information of the organism. The clustered regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated protein-9 nuclease (Cas9) system is a genome editing technique developed by the acquired immune system in the microbes, such as bacteria and archaebacteria, which targets and edits genome sequences according to the principle of complementary base pairing. This technique can be used to edit endogenous genomic DNA sequences in organisms accurately and has been widely used in fields, such as biotechnology, cancer gene therapy, and dermatology. In this review, we summarize the history, structure, mechanism, and application of CRISPR/Cas9 in gene therapy and dermatological diseases.

Keywords: Genome editing, CRISPR/Cas9, gene therapy, dermatology, site mutation, microbes.

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VOLUME: 19
ISSUE: 16
Year: 2019
Page: [1912 - 1919]
Pages: 8
DOI: 10.2174/1871520619666191014103711
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