Induction of Dystrophin Expression by Exon Skipping in mdx Mice Following Intramuscular Injection of Antisense Oligonucleotides Complexed with PEG-PEI Copolymers

Molecular Therapy

Volumn 14, Issue 1, 2006, Pages 88-96

  Williams, Jason H ^a   Sirsi, Shashank R ^a   Latta, Daniel R ^a   Lutz, Gordon J ^a  

^a DREXEL UNIVERSITY COLLEGE OF MEDICINE   (United States)

Author keywords

antisense oligonucleotides; Duchenne muscular dystrophy; dystrophin; exon skipping; mdx mouse; PEG PEI; skeletal muscle

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DRUG CARRIER; DYSTROPHIN; MACROGOL; POLYETHYLENEIMINE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CELL NUCLEUS; CHEMICAL MODIFICATION; CONTROLLED STUDY; CYTOTOXICITY; DRUG EFFICACY; DUCHENNE MUSCULAR DYSTROPHY; EXON; GENE EXPRESSION; GENE INDUCTION; GENE MUTATION; GENETIC TRANSFECTION; IN VIVO STUDY; MALE; MUSCLE CELL; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PARTICLE SIZE; SKELETAL MUSCLE; TREATMENT OUTCOME; WESTERN BLOTTING;

ALTERNATIVE SPLICING; ANIMALS; BASE SEQUENCE; BLOTTING, WESTERN; DYSTROPHIN; EXONS; GENE THERAPY; IMMUNOHISTOCHEMISTRY; INJECTIONS, INTRAMUSCULAR; MICE; MICE, INBRED MDX; MUSCLE FIBERS; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, ANIMAL; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE; POLYETHYLENE GLYCOLS; POLYETHYLENEIMINE; TRANSFECTION;

EID: 33745289016     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2005.11.025     Document Type: Article

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