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Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy

BACKGROUND: Antisense-mediated exon skipping is currently one of the most promising therapeutic approaches for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs) targeting specific exons the DMD reading frame is restored and partially functional dystrophins are produced. Foll...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Aartsma-Rus, Annemieke, Janson, Anneke AM, van Ommen, Gert-Jan B, van Deutekom, Judith CT
Format: Artigo
Sprache:Inglês
Veröffentlicht: BioMed Central 2007
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC1931584/
https://ncbi.nlm.nih.gov/pubmed/17612397
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1471-2350-8-43
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