Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino

Gene Therapy

Volumn 17, Issue 1, 2010, Pages 132-140

  Wu, B ^a   Lu, P ^a   Benrashid, E ^a   Malik, S ^a   Ashar, J ^a   Doran, T J ^a   Lu, Q L ^a  

^a Neuromuscular/ALS Center   (United States)

Author keywords

DMD; Exon skipping; Heart; Morpholino

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CREATINE KINASE; DYSTROPHIN; MORPHOLINE DERIVATIVE; MORPHOLINO OLIGOMER; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; CREATINE KINASE BLOOD LEVEL; DRUG EFFICACY; DRUG MEGADOSE; DUCHENNE MUSCULAR DYSTROPHY; EXON; HEART MUSCLE; HEART MUSCLE CELL; IN VITRO STUDY; IN VIVO STUDY; MOUSE; MYOBLAST; NONHUMAN; OUTCOME ASSESSMENT; PRIORITY JOURNAL; SKELETAL MUSCLE;

ANIMALS; CELLS, CULTURED; CREATINE KINASE; DOSE-RESPONSE RELATIONSHIP, DRUG; DYSTROPHIN; GENE THERAPY; INJECTIONS, INTRAMUSCULAR; MICE; MICE, INBRED C57BL; MICE, INBRED MDX; MORPHOLINES; MUSCULAR DYSTROPHY, ANIMAL; MYOBLASTS, SKELETAL; MYOCARDIUM; MYOCYTES, CARDIAC; OLIGONUCLEOTIDES, ANTISENSE; TRANSFECTION;

MUS;

EID: 74349109205     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2009.120     Document Type: Article

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