Evaluation of 2’-Deoxy-2’-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder typically caused by frame-shifting mutations in the DMD gene. Restoration of the reading frame would allow the production of a shorter but partly functional dystrophin protein as seen in Becker muscular dystrophy patients. This ca...
Huvudupphovsmän: | , , , , , , , |
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Materialtyp: | Artikel |
Språk: | English |
Publicerad: |
Elsevier
2015-01-01
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Serie: | Molecular Therapy: Nucleic Acids |
Ämnen: | |
Länkar: | http://www.sciencedirect.com/science/article/pii/S2162253116300506 |