CRISPR/Cas systems: Delivery and application in gene therapy

Jie Huang, Yitong Zhou, Jie Li*, Aiping Lu*, Chao Liang*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

21 Citations (Scopus)

Abstract

The CRISPR/Cas systems in prokaryotes such as bacteria and archaea are the adaptive immune system to prevent infection from viruses, phages, or other foreign substances. When viruses or phages first invade the bacteria, Cas proteins recognize and cut the DNA from viruses or phages into short fragments that will be integrated into the CRISPR array. Once bacteria are invaded again, the modified CRISPR and Cas proteins react quickly to cut DNA at the specified target location, protecting the host. Due to its high efficiency, versatility, and simplicity, the CRISPR/Cas system has become one of the most popular gene editing technologies. In this review, we briefly introduce the CRISPR/Cas systems, focus on several delivery methods including physical delivery, viral vector delivery, and non-viral vector delivery, and the applications of disease therapy. Finally, some problems in CRISPR/Cas9 technology have been proposed, such as the off-target effects, the efficiency of DNA repair mechanisms, and delivery of CRISPR/Cas system safely and efficiently to the target location.

Original languageEnglish
Article number942325
Number of pages21
JournalFrontiers in Bioengineering and Biotechnology
Volume10
DOIs
Publication statusPublished - 22 Nov 2022

Scopus Subject Areas

  • Biotechnology
  • Bioengineering
  • Histology
  • Biomedical Engineering

User-Defined Keywords

  • CRISPR/Cas systems
  • delivery
  • viral vectors
  • non-viral vectors
  • genome editing
  • gene therapy

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