A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy

Molecular Therapy

Volumn 23, Issue 1, 2015, Pages 192-201

  Mendell, Jerry R ^a,b   Sahenk, Zarife ^a,b   Malik, Vinod ^a   Gomez, Ana M ^a   Flanigan, Kevin M ^a,b   Lowes, Linda P ^b   Alfano, Lindsay N ^b   Berry, Katherine ^b   Meadows, Eric ^a   Lewis, Sarah ^a   Braun, Lyndsey ^a   Shontz, Kim ^a   Rouhana, Maria ^a   Clark, Kelly Reed ^a,b   Rosales, Xiomara Q ^a,b   Al Zaidy, Samiah ^a,b   Govoni, Alessandra ^a   Rodino Klapac, Louise R ^a,b   Hogan, Mark J ^a   Kaspar, Brian K ^a,b  

^a NATIONWIDE CHILDREN S HOSPITAL   (United States)

^b OHIO STATE UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

FOLLISTATIN; PARVOVIRUS VECTOR; DMD PROTEIN, HUMAN; DYSTROPHIN; FOLLISTATIN RELATED PROTEIN; MSTN PROTEIN, HUMAN; MYOSTATIN;

ABSENCE OF SIDE EFFECTS; ADENO ASSOCIATED VIRUS 1; ADULT; ALTERNATIVE RNA SPLICING; ARTICLE; BECKER MUSCULAR DYSTROPHY; BINDING SITE; CELL NUCLEUS; CLINICAL ARTICLE; COHORT ANALYSIS; CYTOMEGALOVIRUS; DRUG MEGADOSE; ENDOMYSIUM; EXERCISE TEST; FOLLISTATIN GENE; GENE THERAPY; HISTOPATHOLOGY; HUMAN; HUMAN CELL; HUMAN TISSUE; LOW DRUG DOSE; MALE; MUSCLE CELL; MUSCLE HYPERTROPHY; MYOFIBROSIS; NUCLEOTIDE SEQUENCE; OUTCOME ASSESSMENT; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PHYSICAL CAPACITY; QUADRICEPS FEMORIS MUSCLE; TREATMENT RESPONSE; VIRAL GENE DELIVERY SYSTEM; WALKING; YOUNG ADULT; ANTAGONISTS AND INHIBITORS; CLINICAL TRIAL; DEFICIENCY; DEPENDOPARVOVIRUS; GENE EXPRESSION; GENE VECTOR; GENETICS; INTRAMUSCULAR DRUG ADMINISTRATION; METABOLISM; MUSCULAR DYSTROPHY, DUCHENNE; PATHOLOGY; PROCEDURES; SKELETAL MUSCLE;

ADENO-ASSOCIATED VIRUS;

ADULT; DEPENDOVIRUS; DYSTROPHIN; FOLLISTATIN-RELATED PROTEINS; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HUMANS; INJECTIONS, INTRAMUSCULAR; MALE; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; MYOSTATIN;

EID: 84920677763     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2014.200     Document Type: Article

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