AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy

Molecular Therapy

Volumn 19, Issue 11, 2011, Pages 2055-2064

  Bortolanza, Sergia ^a   Nonis, Alessandro ^b   Sanvito, Francesca ^c   MacIotta, Simona ^d   Sitia, Giovanni ^c   Wei, Jessica ^e   Torrente, Yvan ^d   Di Serio, Clelia ^b   Chamberlain, Joel R ^e   Gabellini, Davide ^a  

^a DULBECCO TELETHON INSTITUTE   (Italy)

^b VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

^c SAN RAFFAELE HOSPITAL   (Italy)

^d UNIVERSITY OF MILAN   (Italy)

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

Author keywords

[No Author keywords available]

Indexed keywords

FRG1 PROTEIN; GENE PRODUCT; MESSENGER RNA; PARVOVIRUS VECTOR; SHORT HAIRPIN RNA; UNCLASSIFIED DRUG;

ADIPOCYTE; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CHROMOSOME 4Q; CONTROLLED STUDY; DOWN REGULATION; FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY; FRG1 GENE; GENE; GENE DOSAGE; GENE OVEREXPRESSION; HISTOPATHOLOGY; INFLAMMATION; MOUSE; MUSCLE FUNCTION; MYOFIBROSIS; NONHUMAN; RNA INTERFERENCE; TRANSGENIC MOUSE; VIRAL GENE DELIVERY SYSTEM;

MUS MUSCULUS;

EID: 80455173941     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.153     Document Type: Article

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