Skipping to new gene therapies for muscular dystrophy

Nature Medicine

Volumn 9, Issue 8, 2003, Pages 997-998

  Tidball, James G ^a   Spencer, Melissa J ^b  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ANTISENSE OLIGORIBONUCLEOTIDE; DYSTROPHIN; GENTAMICIN; OLIGORIBONUCLEOTIDE; RNA; UNCLASSIFIED DRUG;

ANIMAL MODEL; CHIMERAPLASTY; DUCHENNE MUSCULAR DYSTROPHY; EXON; EXON SKIPPING; GENE MUTATION; GENE THERAPY; MOUSE; MUSCLE FUNCTION; MUSCULAR DYSTROPHY; NONHUMAN; PRIORITY JOURNAL; SHORT SURVEY; TECHNIQUE;

NON-PROGRAMMATIC;

ANIMALS; DISEASE MODELS, ANIMAL; EXONS; GENE THERAPY; HUMANS; MICE; MUSCULAR DYSTROPHY, ANIMAL; MUSCULAR DYSTROPHY, DUCHENNE; OLIGORIBONUCLEOTIDES, ANTISENSE; RNA; RNA SPLICING;

ANIMALIA;

EID: 0041965948     PISSN: 10788956     EISSN: None     Source Type: Journal    
DOI: 10.1038/nm0803-997     Document Type: Short Survey

References (11)

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2. Lu, Q.L. et al. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Nat. Med. 9, 1009-1014 (2003).
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11. Wagner, K. R. et al. Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations. Ann. Neurol. 49, 706-711 (2001)