Unraveling the potential of CRISPR-Cas9 for gene therapy.
Expert Opin Biol Ther
; 15(3): 311-4, 2015 Mar.
Article
en En
| MEDLINE
| ID: mdl-25535790
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.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Terapia Genética
/
Sistemas CRISPR-Cas
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Expert Opin Biol Ther
Asunto de la revista:
BIOLOGIA
/
TERAPEUTICA
Año:
2015
Tipo del documento:
Article