Application of CRISPR-Cas system in gene therapy: Pre-clinical progress in animal model

DNA Repair (Amst). 2016 Oct:46:1-8. doi: 10.1016/j.dnarep.2016.07.004. Epub 2016 Jul 30.

Abstract

The clustered regularly interspaced short palindromic repeats (CRISPRs) and their associated proteins (Cas) belong to the crucial adaptive immune system, which exist in archaea and bacteria. Currently, CRISPR-Cas9 system has been modified and widely used to edit genome. In this review, we summarized the discovery, classification and mechanism of CRISPR-Cas system and further discussed the application of CRISPR-Cas9 in gene therapy, mainly in disease models.

Keywords: CRISPR-Cas9; DNA repair; Disease model; Gene therapy; Genome editing.

Publication types

  • Review

MeSH terms

  • Acquired Immunodeficiency Syndrome / genetics
  • Acquired Immunodeficiency Syndrome / pathology
  • Acquired Immunodeficiency Syndrome / therapy*
  • Adenocarcinoma / genetics
  • Adenocarcinoma / pathology
  • Adenocarcinoma / therapy*
  • Adenocarcinoma of Lung
  • Animals
  • CRISPR-Cas Systems*
  • Cataract / genetics
  • Cataract / pathology
  • Cataract / therapy*
  • Disease Models, Animal
  • Gene Editing / methods
  • Genetic Therapy / methods
  • Humans
  • Lung Neoplasms / genetics
  • Lung Neoplasms / pathology
  • Lung Neoplasms / therapy*
  • Mice
  • Muscular Dystrophy, Duchenne / genetics
  • Muscular Dystrophy, Duchenne / pathology
  • Muscular Dystrophy, Duchenne / therapy*
  • Obesity / genetics
  • Obesity / pathology
  • Obesity / therapy*
  • Transcription Activator-Like Effector Nucleases / genetics
  • Transcription Activator-Like Effector Nucleases / metabolism
  • Tyrosinemias / genetics
  • Tyrosinemias / pathology
  • Tyrosinemias / therapy*

Substances

  • Transcription Activator-Like Effector Nucleases