CRISPR/dCas9-mediated transcriptional inhibition ameliorates the epigenetic dysregulation at D4Z4 and represses DUX4-fl in FSH muscular dystrophy

Molecular Therapy

Volumn 24, Issue 3, 2016, Pages 527-535

  Himeda, Charis L ^a   Jones, Takako I ^a   Jones, Peter L ^a  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

MESSENGER RNA; DUX4 PROTEIN, HUMAN; HOMEODOMAIN PROTEIN; HYBRID PROTEIN; MICROSATELLITE DNA; PROTEIN BINDING;

ARTICLE; CONTROLLED STUDY; CRISPR CAS SYSTEM; DOWN REGULATION; DUX4 GENE; EPIGENETICS; EXON; FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY; GENE CONTROL; GENE LOCUS; GENE REPRESSION; GENE TARGETING; HUMAN; HUMAN CELL; HUMAN TISSUE; MARKER GENE; MUSCLE CELL; PROMOTER REGION; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; GENE EDITING; GENE EXPRESSION REGULATION; GENETIC EPIGENESIS; GENETIC TRANSCRIPTION; GENETICS; METABOLISM; TRANSCRIPTION INITIATION;

CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS; CRISPR-CAS SYSTEMS; EPIGENESIS, GENETIC; EXONS; GENE EDITING; GENE EXPRESSION REGULATION; GENE TARGETING; GENETIC LOCI; HOMEODOMAIN PROTEINS; HUMANS; MICROSATELLITE REPEATS; MUSCLE CELLS; MUSCULAR DYSTROPHY, FACIOSCAPULOHUMERAL; PROMOTER REGIONS, GENETIC; PROTEIN BINDING; RECOMBINANT FUSION PROTEINS; TRANSCRIPTION, GENETIC; TRANSCRIPTIONAL ACTIVATION;

EID: 84960412514     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.200     Document Type: Article

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