title: Unraveling the potential of CRISPR-Cas9 for gene therapy.
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.
authors.affiliation: North Carolina State University, Department of Food, Bioprocessing and Nutrition Sciences , Raleigh, NC 27695 , USA +1 919 513 1644 ; rbarran@ncsu.edu.
authors.forename: Rodolphe
authors.initials: R
authors.lastname: Barrangou
authors.forename: Andrew P
authors.initials: AP
authors.lastname: May
mesh_terms: D000818:Animals; D064113:CRISPR-Cas Systems / Q000235:genetics*; D004260:DNA Repair / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods / Q000639:trends*; D016678:Genome; D006801:Humans
keywords: CRISPR; Cas9; gene therapy; genome editing