New insights in gene-derived therapy: The example of Duchenne muscular dystrophy

Annals of the New York Academy of Sciences

Volumn 1214, Issue 1, 2010, Pages 199-212

  Aartsma Rus, Annemieke ^a   Den Dunnen, Johan T ^a   Van Ommen, Gert Jan B ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

Duchenne muscular dystrophy; Exon skipping; Forced read through; Monogenic diseases; Personalized medicine

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ATALUREN; DYSTROPHIN; GENE PRODUCT; GENTAMICIN;

ARTICLE; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; DRUG TOLERABILITY; DUCHENNE MUSCULAR DYSTROPHY; EXON; GENE MUTATION; GENE THERAPY; HUMAN; LOW DRUG DOSE; NEPHROTOXICITY; NONHUMAN; OTOTOXICITY; STOP CODON; TREATMENT OUTCOME;

EID: 78650397994     PISSN: 00778923     EISSN: 17496632     Source Type: Book Series    
DOI: 10.1111/j.1749-6632.2010.05836.x     Document Type: Article

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