Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides

Molecular Therapy

Volumn 10, Issue 2, 2004, Pages 232-240

  Bremmer Bout, Mattie ^a   Aartsma Rus, Annemieke ^a   de Meijer, Emile J ^a   Kaman, Wendy E ^a   Janson, Anneke A M ^a   Vossen, Rolf H A M ^a   van Ommen, Gert Jan B ^a   den Dunnen, Johan T ^a   van Deutekom, Judith C T ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

Antisense oligonucleotides; Duchenne muscular dystrophy; Dystrophin; Exon skipping; hDMD mice; MALDI TOF; Polyethylenimine

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; POLYETHYLENEIMINE; REAGENT;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CORRELATION FUNCTION; DATA ANALYSIS; DRUG EFFICACY; DRUG MECHANISM; DRUG SCREENING; DUCHENNE MUSCULAR DYSTROPHY; EXON; GENE DELIVERY SYSTEM; GENE SEQUENCE; GENE TARGETING; GENE THERAPY; IN VIVO STUDY; MOUSE; MUSCLE CELL; MUSCLE REGENERATION; NONHUMAN; TRANSGENIC MOUSE; WILD TYPE;

ANIMALS; DISEASE MODELS, ANIMAL; DRUG EVALUATION, PRECLINICAL; DYSTROPHIN; EXONS; GENE TARGETING; HUMANS; MICE; MICE, TRANSGENIC; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE; RNA, MESSENGER;

ANIMALIA; MUS MUSCULUS;

EID: 4344693568     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2004.05.031     Document Type: Article

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