Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy

Human Molecular Genetics

Volumn 24, Issue 17, 2015, Pages 4971-4983

  Bisset, Darren R ^a   Stepniak Konieczna, Ewa A ^a   Zavaljevski, Maja ^a   Wei, Jessica ^a   Carter, Gregory T ^a   Weiss, Michael D ^b   Chamberlain, Joel R ^a  

^a UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF WASHINGTON   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

MESSENGER RNA; MICRORNA; PARVOVIRUS VECTOR; DNA BINDING PROTEIN; MBNL1 PROTEIN, MOUSE; RNA BINDING PROTEIN; SMALL INTERFERING RNA;

3' UNTRANSLATED REGION; ALPHA SKELETAL MUSCLE ACTIN GENE; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DMPK GENE; GENE; GENE CASSETTE; GENE EXPRESSION; MOUSE; MUSCLE HYPERTROPHY; MYOTONIC DYSTROPHY; NONHUMAN; PRIORITY JOURNAL; RNA INTERFERENCE; RNA SPLICING; SYSTEMIC THERAPY; VIRAL GENE THERAPY; VIRUS RECOMBINANT; ADMINISTRATION AND DOSAGE; ANIMAL; CELL LINE; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE ORDER; GENE SILENCING; GENE THERAPY; GENE VECTOR; GENETIC TRANSCRIPTION; GENETIC TRANSDUCTION; GENETICS; HUMAN; METABOLISM; PATHOLOGY; PHENOTYPE; SKELETAL MUSCLE; TRANSGENE; TRANSGENIC MOUSE;

ANIMALS; CELL LINE; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DNA-BINDING PROTEINS; GENE EXPRESSION; GENE ORDER; GENE SILENCING; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MICE; MICE, TRANSGENIC; MUSCLE, SKELETAL; MYOTONIC DYSTROPHY; PHENOTYPE; RNA INTERFERENCE; RNA SPLICING; RNA, SMALL INTERFERING; RNA-BINDING PROTEINS; TRANSCRIPTION, GENETIC; TRANSDUCTION, GENETIC; TRANSGENES;

EID: 84941930816     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddv219     Document Type: Article

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