Genetic therapies: Correcting genetic defects with CRISPR-Cas9

Nature Reviews Genetics

Volumn 15, Issue 2, 2014, Pages 63-

  Lokody, Isabel ^a  

^a NONE

Author keywords

[No Author keywords available]

Indexed keywords

CRISPR ASSOCIATED PROTEIN; GUIDE RNA; OLIGONUCLEOTIDE; PROTEIN CRISPR CAS9; UNCLASSIFIED DRUG;

ALLELE; CFTR GENE; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; DNA END JOINING REPAIR; DNA STRAND BREAKAGE; GENE MUTATION; GENE THERAPY; GENETIC DISORDER; GENETIC RECOMBINATION; GENETIC TRANSFECTION; HETEROZYGOTE; HUMAN; INTRON; NONHUMAN; NOTE; PRIORITY JOURNAL; REGULATOR GENE;

EID: 84892824264     PISSN: 14710056     EISSN: 14710064     Source Type: Journal    
DOI: 10.1038/nrg3656     Document Type: Note

References (2)

1. ORIGINAL RESEARCH PAPERS. Schwank, G. et al. Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients. Cell stem cell 13, 653-658 (2013)
2. Wu, Y. et al. Correction of a genetic disease in mouse via use of CRISPR-Cas9. Cell stem cell 13, 659-662 (2013).