RNAi-mediated gene silencing of mutant myotilin improves myopathy in LGMD1A mice

Molecular Therapy Nucleic Acids

Volumn 3, Issue , 2014, Pages e160-

  Liu, Jian ^a   Wallace, Lindsay M ^a   Garwick Coppens, Sara E ^a   Sloboda, Darcée D ^b   Davis, Carol S ^b   Hakim, Chady H ^c   Hauser, Michael A ^d   Brooks, Susan V ^b   Mendell, Jerry R ^a,e   Harper, Scott Q ^a,e  

^a THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN S HOSPITAL   (United States)

^b UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^c University of Missouri   (United States)

^d DUKE UNIVERSITY MEDICAL CENTER   (United States)

^e THE OHIO STATE UNIVERSITY COLLEGE OF MEDICINE   (United States)

Author keywords

AAV; Dominant muscular dystrophy; LGMD1A; Myofibrillar myopathy; Myotilin; RNAi therapy

Indexed keywords

MICRORNA; PARVOVIRUS VECTOR; PROTEIN AGGREGATE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; GASTROCNEMIUS MUSCLE; GENE; GENE SILENCING; HISTOPATHOLOGY; LIMB GIRDLE MUSCULAR DYSTROPHY; MALE; MOLECULAR THERAPY; MOUSE; MUSCLE MASS; MYOFIBRILLAR MYOPATHY; MYOPATHY; MYOTILIN GENE; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; RNA INTERFERENCE; VIRAL GENE THERAPY;

EID: 84902983525     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2014.13     Document Type: Article

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