Allele-specific gene silencing of mutant mRNA restores cellular function in ullrich congenital muscular dystrophy fibroblasts

Molecular Therapy Nucleic Acids

Volumn 3, Issue , 2014, Pages

  Noguchi, Satoru ^a,b   Ogawa, Megumu ^a   Kawahara, Genri ^a,c   Malicdan, May Christine ^a,d   Nishino, Ichizo ^a,b  

^a NATIONAL INSTITUTE OF NEUROSCIENCE   (Japan)

^b NATIONAL INSTITUTE OF MENTAL HEALTH   (Japan)

^c BOSTON CHILDREN S HOSPITAL   (United States)

^d NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

Author keywords

allele specific; autosomal dominant inheritance; Co16a1; gene silencing; muscular dystrophy; siRNA

Indexed keywords

COLLAGEN TYPE 6; MESSENGER RNA; SMALL INTERFERING RNA; COLLAGEN TYPE 6 A1; UNCLASSIFIED DRUG;

ALLELE; ARTICLE; AUTOSOMAL DOMINANT INHERITANCE; CELL FUNCTION; CONTROLLED STUDY; FIBROBLAST; GENE SILENCING; HUMAN; HUMAN CELL; MUSCULAR DYSTROPHY; MUTANT; PHENOTYPE; POINT MUTATION; PRIORITY JOURNAL; ULLRICH CONGENITAL MUSCULAR DYSTROPHY; WILD TYPE; 3T3 CELL LINE; CELL CULTURE; GENE REPRESSION; GENETIC TRANSCRIPTION; HUMAN TISSUE; LUCIFERASE ASSAY; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; VIRAL GENE DELIVERY SYSTEM;

EID: 84903462621     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2014.22     Document Type: Article

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