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...

Full description

Saved in:
Bibliographic Details
Main Authors: 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
Language:Inglês
Published: The American Society of Human Genetics 2004
Subjects:
Articles
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC1181915/
https://ncbi.nlm.nih.gov/pubmed/14681829
Tags: Add Tag
No Tags, Be the first to tag this record!

Similar Items