In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse

Molecular Therapy

Volumn 18, Issue 11, 2010, Pages 1995-2005

  Aoki, Yoshitsugu ^a,b   Nakamura, Akinori ^a   Yokota, Toshifumi ^a,c   Saito, Takashi ^a,d   Okazawa, Hitoshi ^b   Nagata, Tetsuya ^a   Takeda, Shin'Ichi ^a  

^a NATIONAL INSTITUTE OF NEUROSCIENCE   (Japan)

^b TOKYO MEDICAL AND DENTAL UNIVERSITY   (Japan)

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

^d TOKYO WOMEN'S MEDICAL UNIVERSITY   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; PHOSPHORODIAMIDATE MORPHOLINO OLIGOMER; UNCLASSIFIED DRUG;

3' UNTRANSLATED REGION; 5' UNTRANSLATED REGION; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTISENSE THERAPY; ARTICLE; CONTROLLED STUDY; DRUG DESIGN; DRUG DOSE ESCALATION; EXON; EXON SKIPPING; FUNCTIONAL ASSESSMENT; GENE DELETION; GENE TARGETING; HINGE REGION; MALE; MOUSE; MUSCLE FUNCTION; MUSCULAR DYSTROPHY; NONHUMAN; OPTIMAL DRUG DOSE; PROTEIN EXPRESSION; PROTEIN FUNCTION; PROTEIN STRUCTURE; RNA SPLICING; SINGLE DRUG DOSE; WILD TYPE; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

MUS;

EID: 78049472917     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2010.186     Document Type: Article

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