Reading frame correction by targeted genome editing restores dystrophin expression in cells from duchenne muscular dystrophy patients

Molecular Therapy

Volumn 21, Issue 9, 2013, Pages 1718-1726

  Ousterout, David G ^a   Perez Pinera, Pablo ^a   Thakore, Pratiksha I ^a   Kabadi, Ami M ^a   Brown, Matthew T ^a   Qin, Xiaoxia ^b   Fedrigo, Olivier ^b   Mouly, Vincent ^c   Tremblay, Jacques P ^d   Gersbach, Charles A ^a,b,e  

^a Duke University   (United States)

^b DUKE UNIVERSITY   (United States)

^c SORBONNE UNIVERSITÉ   (France)

^d LAVAL UNIVERSITY   (Canada)

^e DUKE UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN; MYOD PROTEIN; NUCLEASE; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; UNCLASSIFIED DRUG;

ARTICLE; CELL LINEAGE; CELL STRAIN HEK293; COMPUTER ANALYSIS; CONTROLLED STUDY; DNA MODIFICATION; DRUG MECHANISM; DUCHENNE MUSCULAR DYSTROPHY; EXOME; EXON; FRAMESHIFT MUTATION; GENE EXPRESSION; GENE LOCUS; GENE MUTATION; GENE TARGETING; GENETIC ENGINEERING; GENOME EDITING; HUMAN; HUMAN CELL; MYOBLAST; NUCLEAR REPROGRAMMING; NUCLEOTIDE SEQUENCE; PROTEIN EXPRESSION; SEQUENCE ANALYSIS; SKIN FIBROBLAST; STOP CODON;

DYSTROPHIN; ENDONUCLEASES; EXOME; GENE TARGETING; GENETIC THERAPY; GENOME, HUMAN; HEK293 CELLS; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; READING FRAMES;

EID: 84883743140     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.111     Document Type: Article

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