Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

Proceedings of the National Academy of Sciences of the United States of America

Volumn 109, Issue 34, 2012, Pages 13763-13768

  Aoki, Yoshitsugu ^a   Yokota, Toshifumi ^a,b,c   Nagata, Tetsuya ^a   Nakamura, Akinori ^a,d   Tanihata, Jun ^a   Saito, Takashi ^a,e   Duguez, Stephanie M R ^b,f   Nagaraju, Kanneboyina ^b,g   Hoffman, Eric P ^b,g   Partridge, Terence ^b,g   Takeda, Shin'ichi ^a  

^a NATIONAL INSTITUTE OF NEUROSCIENCE   (Japan)

^b CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^c UNIVERSITY OF ALBERTA   (Canada)

^d SHINSHU UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^e TOKYO WOMEN'S MEDICAL UNIVERSITY   (Japan)

^f SORBONNE UNIVERSITÉ   (France)

^g GEORGE WASHINGTON UNIVERSITY SCHOOL OF MEDICINE AND HEALTH SCIENCES   (United States)

Author keywords

Gene therapy; Molecular therapy; Nucleic acid therapy; Oligonucleotides; Personalized medicine

Indexed keywords

ANTISENSE OLIGOMORPHOLINO; DYSTROPHIN; MDX52 PROTEIN; MORPHOLINE DERIVATIVE; PROTEIN; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; EXON; FEASIBILITY STUDY; GENE DELETION; HISTOPATHOLOGY; IN VITRO STUDY; MALE; MOUSE; MUSCLE STRENGTH; MYOTUBE; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; SKELETAL MUSCLE; WILD TYPE;

ANIMALS; DYSTROPHIN; EXONS; GENE DELETION; GENE THERAPY; INDIVIDUALIZED MEDICINE; MALE; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; MICE, TRANSGENIC; MODELS, GENETIC; MUSCLE FIBERS, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE;

EID: 84865288612     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1204638109     Document Type: Article

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