An introduction to CRISPR-Cas systems for reprogramming the genome of mammalian cells

Prog Mol Biol Transl Sci. 2021:181:1-13. doi: 10.1016/bs.pmbts.2021.01.010. Epub 2021 Feb 22.

Abstract

In the past few decades, it has been possible to introduce unprecedented mutations in genes of the mammalian cells owing to the development of advanced technologies/methods/assays. Sometimes, these mutations occurring at the cellular level may even cost the life of organisms. A number of diseases in mammals have shown to leave some serious impact on their lives. There are no drugs or medicines available in market for the correction or repair of these mutated genes in order to reverse gene function. A pressing need therefore arises to develop a next generation technology that cannot just corrects gene mutations but also restores gene function. Recent advances in CRISPR-Cas9 technology play a key role for correction of defective genes in wide range of mammalian cells. This chapter highlights CRISPR-Cas systems for basic, biomedical, biotechnological and therapeutic applications.

Keywords: CRISPR-Cas systems; Diseases; Drug; Knock in; Knock out; Mutations; Therapeutic.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • CRISPR-Cas Systems* / genetics
  • Gene Editing*
  • Mutation
  • Phenotype