Use of CRISPR/Cas9 to model brain diseases

Prog Neuropsychopharmacol Biol Psychiatry. 2018 Feb 2;81:488-492. doi: 10.1016/j.pnpbp.2017.04.003. Epub 2017 Apr 6.


Aging-related brain diseases consist of a number of important neurodegenerative disorders, including Alzheimer's, Parkinson's, and Huntington's diseases, all of which have become more prevalent as the life expectancy of humans is prolonged. Age-dependent brain disorders are associated with both environmental insults and genetic mutations. For those brain disorders that are inherited, gene editing is an important tool for establishing animal models to investigate the pathogenesis of disease and identify effective treatments. Here we focus on the tools for gene editing, especially CRISPR/Cas9, and discuss their application for generating animal models that can recapitulate the brain pathology seen in human diseases. We also highlight the advantages and disadvantages of establishing genetically modified animal models. Finally, we discuss recent findings to resolve technical issues related to the use of CRISPR/Cas9 for generating animal models of brain diseases.

Keywords: Animal models; CRISPR/Cas9; Gene editing; Neurodegeneration.

Publication types

  • Review

MeSH terms

  • Animals
  • Brain / pathology
  • Brain Diseases* / genetics
  • Brain Diseases* / pathology
  • CRISPR-Cas Systems*
  • Disease Models, Animal*
  • Gene Editing / methods*