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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Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: The American Society of Human Genetics 2004
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC1181915/
https://ncbi.nlm.nih.gov/pubmed/14681829
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