Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials

Neuromuscular Disorders

Volumn 20, Issue 2, 2010, Pages 102-110

  Popplewell, Linda J ^a   Adkin, Carl ^b   Arechavala Gomeza, Virginia ^b   Aartsma Rus, Annemieke ^c   de Winter, Christa L ^c   Wilton, Steve D ^d   Morgan, Jennifer E ^b   Muntoni, Francesco ^b   Graham, Ian R ^a   Dickson, George ^a  

^a UNIVERSITY OF LONDON   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^d UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

Author keywords

Antisense oligonucleotides; Duchenne muscular dystrophy; Exon skipping; Phosphorodiamidate morpholino oligomer

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DUCHENNE MUSCULAR DYSTROPHY PROTEIN; DYSTROPHIN; MUSCLE PROTEIN; OLIGONUCLEOTIDE; PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DOSE RESPONSE; DRUG EFFICACY; DRUG MECHANISM; DRUG SCREENING; DUCHENNE MUSCULAR DYSTROPHY; EXON; GENE DELETION; GENE TARGETING; HUMAN; HUMAN CELL; HUMAN TISSUE; IN VITRO STUDY; IN VIVO STUDY; MOUSE; MUSCLE BIOPSY; MUSCLE CELL; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; PROTEIN EXPRESSION; SEQUENCE ANALYSIS; SINGLE DRUG DOSE; SKELETAL MUSCLE; TRANSGENIC MOUSE;

ANIMALS; BASE SEQUENCE; CELLS, CULTURED; DISEASE MODELS, ANIMAL; DYSTROPHIN; EXONS; GENE EXPRESSION REGULATION; GENE TARGETING; GENE THERAPY; HUMANS; MALE; MICE; MICE, TRANSGENIC; MORPHOLINES; MUSCULAR DYSTROPHY, DUCHENNE; MUTATION; OLIGONUCLEOTIDES, ANTISENSE; TRANSCRIPTION, GENETIC;

EID: 75149113504     PISSN: 09608966     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.nmd.2009.10.013     Document Type: Article

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