CRISPR/Cas9 for genome editing: progress, implications and challenges

Hum Mol Genet. 2014 Sep 15;23(R1):R40-6. doi: 10.1093/hmg/ddu125. Epub 2014 Mar 20.

Abstract

Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) protein 9 system provides a robust and multiplexable genome editing tool, enabling researchers to precisely manipulate specific genomic elements, and facilitating the elucidation of target gene function in biology and diseases. CRISPR/Cas9 comprises of a nonspecific Cas9 nuclease and a set of programmable sequence-specific CRISPR RNA (crRNA), which can guide Cas9 to cleave DNA and generate double-strand breaks at target sites. Subsequent cellular DNA repair process leads to desired insertions, deletions or substitutions at target sites. The specificity of CRISPR/Cas9-mediated DNA cleavage requires target sequences matching crRNA and a protospacer adjacent motif locating at downstream of target sequences. Here, we review the molecular mechanism, applications and challenges of CRISPR/Cas9-mediated genome editing and clinical therapeutic potential of CRISPR/Cas9 in future.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics*
  • DNA Cleavage
  • Gene Expression Regulation
  • Gene Transfer Techniques
  • Genetic Therapy
  • Genome
  • Genomics / methods*
  • Humans
  • Protein Structure, Tertiary
  • RNA Editing / genetics*
  • Sequence Analysis, DNA