Antisense-Induced Multiexon Skipping for Duchenne Muscular Dystrophy Makes More Sense

Dystrophin deficiency, which leads to severe and progressive muscle degeneration in patients with Duchenne muscular dystrophy (DMD), is caused by frameshifting mutations in the dystrophin gene. A relatively new therapeutic strategy is based on antisense oligonucleotides (AONs) that induce the specif...

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Auteurs principaux: Aartsma-Rus, Annemieke, Janson, Anneke A. M., Kaman, Wendy E., Bremmer-Bout, Mattie, van Ommen, Gert-Jan B., den Dunnen, Johan T., van Deutekom, Judith C. T.
Format: Artigo
Langue:Inglês
Publié: The American Society of Human Genetics 2004
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Articles
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC1181915/
https://ncbi.nlm.nih.gov/pubmed/14681829
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