Unraveling the potential of CRISPR-Cas9 for gene therapy

Rodolphe Barrangou, Andrew P. May

Research output: Contribution to journalReview articlepeer-review

21 Scopus citations

Abstract

The molecular machinery from the prokaryotic clustered regularly interspaced short palindromic repeats (CRISPR)-Cas immune system has broadly been repurposed for genome editing in eukaryotes. In particular, the sequence-specific Cas9 endonuclease can be flexibly harnessed for the genesis of precise double-stranded DNA breaks, using single guide RNAs that are readily programmable. The endogenous DNA repair machinery subsequently generates genome modifications, either by random insertion or deletions using non-homologous end joining (NHEJ), or designed integration of mutations or genetic material using homology-directed repair (HDR) templates. This technology has opened new avenues for the investigation of genetic diseases in general, and for gene therapy applications in particular.

Original languageEnglish (US)
Pages (from-to)311-314
Number of pages4
JournalExpert Opinion on Biological Therapy
Volume15
Issue number3
DOIs
StatePublished - Mar 1 2015

All Science Journal Classification (ASJC) codes

  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry

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