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Anticancer Agents Med Chem. 2019 Oct 13. doi: 10.2174/1871520619666191014103711. [Epub ahead of print]

CRISPR/Cas9 System and its Research Progress in Gene Therapy.

Author information

1
Department of Oncology, Affiliated Hospital of Xuzhou Medical University, Xuzhou 221002. China.
2
Department of Dermatology, The Affiliated Huai'an Hospital of Xuzhou Medical University, The Second People's Hospital of Huai'an, No.62, Huaihai Road(S.), Huai'an 223002. China.
3
Department of Dermatology, Affiliated Hospital of Xuzhou Medical University, Xuzhou 221002. China.

Abstract

Genome editing technology 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 genomic editing technique developed by the acquired immune system in 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:

CRISPR/Cas9; Genome editing; dermatology; gene therapy; microbes; site mutation

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