Molecular Therapy: Nucleic Acids (Jan 2016)

CRISPR-Cas9 System as a Versatile Tool for Genome Engineering in Human Cells

  • Xuelian Wang,
  • Xiumin Huang,
  • Xiuli Fang,
  • Youzhong Zhang,
  • Wanpeng Wang

DOI
https://doi.org/10.1038/mtna.2016.95
Journal volume & issue
Vol. 5, no. C

Abstract

Read online

Targeted nucleases are influential instruments for intervening in genome revision with great accuracy. RNA-guided Cas9 nucleases produced from clustered regularly interspaced short palindromic repeats (CRISPR)-Cas systems have noticeably altered the means to modify the genomes of distinct organisms. They can be notably used to facilitate effective genome manipulation in eukaryotic cells by clearly detailing a 20-nt targeting sequence inside its directed RNA. We discuss the most recent advancements in the molecular basis of the type II CRISPR/Cas system and encapsulate applications and elements affecting its use in human cells. We also propose possible applications covering its uses ranging from basic science to implementation in the clinic.

Keywords