The muscular dystrophies: Distinct pathogenic mechanisms invite novel therapeutic approaches

Current Rheumatology Reports

Volumn 13, Issue 3, 2011, Pages 199-207

  Sahenk, Zarife ^a   Mendell, Jerry R ^a  

^a OHIO STATE UNIVERSITY   (United States)

Author keywords

Aminoglycosides; D4Z4; DM1; DM2; DMD; DMPK; DUX4; Dystrophin; Exon skipping; Facioscapulohumeral muscular dystrophy; Gene therapy; Gentamicin; Muscular dystrophy; Mutation suppression; Myotonic muscular dystrophy; ZNF9

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; ETEPLIRSEN; GENOMIC DNA; PARVOVIRUS VECTOR; PRO 051; UNCLASSIFIED DRUG;

ARTICLE; AUTOSOMAL DOMINANT INHERITANCE; DISEASE SEVERITY; DMD GENE; DUCHENNE MUSCULAR DYSTROPHY; DUX4 GENE; EXON SKIPPING; FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY; GENE; GENE MUTATION; GENE REPLACEMENT THERAPY; GENETIC LINKAGE; HUMAN; MOLECULAR CLONING; MYOTONIC DYSTROPHY; NONHUMAN; PATHOGENESIS; PHENOTYPE; PREVALENCE; RNA INTERFERENCE; STOP CODON;

GENE EXPRESSION PROFILING; GENE THERAPY; HOMEODOMAIN PROTEINS; HUMANS; MUSCLE, SKELETAL; MUSCULAR DYSTROPHIES; RNA, SMALL INTERFERING;

EID: 79957512849     PISSN: 15233774     EISSN: 15346307     Source Type: Journal    
DOI: 10.1007/s11926-011-0178-6     Document Type: Article

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