The therapeutic potential of antisense-mediated exon skipping

Current Opinion in Molecular Therapeutics

Volumn 10, Issue 2, 2008, Pages 140-149

  Van Ommen, G J ^a   Van Deutekom, J ^b   Aartsma Rus, A ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^b Prosensa BV   (Netherlands)

Author keywords

Antisense oligonucleotide; Duchenne muscular dystrophy; Exon skipping; Genetic disease; Splicing modulation; Therapy

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; APOLIPOPROTEIN B100; ATM PROTEIN; DYSTROPHIN; MESSENGER RNA; PARVOVIRUS VECTOR; PRO 051; SMALL NUCLEAR RIBONUCLEOPROTEIN; UNCLASSIFIED DRUG; UTROPHIN;

ATAXIA TELANGIECTASIA; BECKER MUSCULAR DYSTROPHY; BINDING SITE; CLINICAL TRIAL; DISEASE SEVERITY; DUCHENNE MUSCULAR DYSTROPHY; EXON; GENE MUTATION; HUMAN; INTRON; NONHUMAN; PHENOTYPE; PROTEIN BINDING; REVIEW; RNA SPLICING; SINGLE DRUG DOSE;

ALTERNATIVE SPLICING; CLINICAL TRIALS AS TOPIC; EXONS; GENETIC VECTORS; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE;

EID: 41849104182     PISSN: 14648431     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

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