Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of α1-syntrophin and α-dystrobrevin in skeletal muscles of mdx mice

Human Gene Therapy

Volumn 22, Issue 11, 2011, Pages 1379-1388

  Koo, Taeyoung ^a   Malerba, Alberto ^a   Athanasopoulos, Takis ^a   Trollet, Capucine ^b,c,d,e   Boldrin, Luisa ^f   Ferry, Arnaud ^b,c,d,e,g   Popplewell, Linda ^a   Foster, Helen ^a   Foster, Keith ^a   Dickson, George ^a  

^a UNIVERSITY OF LONDON   (United Kingdom)

^b INSERM   (France)

^c CNRS   (France)

^d Institut de Myologie   (France)

^e SORBONNE UNIVERSITÉ   (France)

^f UNIVERSITY COLLEGE LONDON   (United Kingdom)

^g UNIVERSITÉ PARIS DESCARTES   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA 1 SYNTROPHIN; ALPHA DYSTROBREVIN; DYSTROBREVIN; DYSTROPHIN; MICRODYSTROPHIN; PARVOVIRUS VECTOR; SYNTROPHIN; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BINDING SITE; CARBOXY TERMINAL SEQUENCE; CONTROLLED STUDY; CYTOSKELETON; EXTRACELLULAR MATRIX; GENE DELETION; GENE EXPRESSION; HISTOPATHOLOGY; MOUSE; MUSCLE CELL; MUSCLE CONTRACTION; MUSCLE INJURY; NONHUMAN; SARCOLEMMA; TIBIALIS ANTERIOR MUSCLE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; WESTERN BLOTTING;

AMINO ACID MOTIFS; ANIMALS; CALCIUM-BINDING PROTEINS; DEPENDOVIRUS; DYSTROPHIN; DYSTROPHIN-ASSOCIATED PROTEINS; GENE THERAPY; GENETIC VECTORS; HEK293 CELLS; HUMANS; MEMBRANE PROTEINS; MICE; MICE, INBRED MDX; MUSCLE PROTEINS; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, ANIMAL; MUSCULAR DYSTROPHY, DUCHENNE;

ADENO-ASSOCIATED VIRUS; MUS;

EID: 81855224742     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2011.020     Document Type: Article

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