The CRISPR way to think about Duchenne's

New England Journal of Medicine

Volumn 374, Issue 17, 2016, Pages 1684-1686

  Calos, Michele P ^a  

^a STANFORD UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; CAS9 PROTEIN; DYSTROPHIN; ENDONUCLEASE; GUIDE RNA; MESSENGER RNA; MICRODYSTROPHIN; OLIGONUCLEOTIDE; UNCLASSIFIED DRUG;

ALTERNATIVE RNA SPLICING; CRISPR CAS SYSTEM; DMD GENE; DNA SEQUENCE; DUCHENNE MUSCULAR DYSTROPHY; ELECTROPORATION; EXON; GENE; GENE MUTATION; GENE VECTOR; GENETIC ENGINEERING; HUMAN; IMMUNE RESPONSE; INTRON; MUSCLE CELL; MUSCLE FUNCTION; MUSCLE STEM CELL; NONHUMAN; NONSENSE MUTATION; OPEN READING FRAME; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN SYNTHESIS; PROTEIN SYNTHESIS INHIBITION; REVIEW; RNA TRANSLATION; VIRAL GENE DELIVERY SYSTEM; ADENOVIRIDAE; ANIMAL; BIOSYNTHESIS; DISEASE MODEL; GENETICS; MOUSE; MUTATION; PROCEDURES; SKELETAL MUSCLE;

ADENOVIRIDAE; ANIMALS; CRISPR-CAS SYSTEMS; DISEASE MODELS, ANIMAL; DYSTROPHIN; GENETIC ENGINEERING; GENETIC VECTORS; HUMANS; MICE; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; PROTEIN BIOSYNTHESIS; RNA, MESSENGER;

EID: 84964988234     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMcibr1601383     Document Type: Review

References (5)

1. Long C, Amoasii L, Mireault AA, et al. Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science 2016; 351: 400-3.
2. Nelson CE, Hakim CH, Ousterout DG, et al. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science 2016; 351: 403-7.
3. Tabebordbar M, Zhu K, Cheng JK, et al. In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science 2016; 351: 407-11.
4. Xu L, Park KH, Zhao L, et al. CRISPR-mediated genome editing restores dystrophin expression and function in mdx mice. Mol Ther 2016; 24: 564-9.
5. Gregorevic P, Allen JM, Minami E, et al. rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med 2006; 12: 787-9.