Challenges for antisense oligonucleotide-based therapeutics, in particular for exon 51-skipping in Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a lethal muscle disorder characterized by mutations in the DMD gene. These mutations primarily disrupt the reading frame, leading to an absence of functional dystrophin protein. Exon-skipping through the use of antisense oligonucleotides has served as a promising...
| Autori principali: | , , , |
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| Natura: | Journal article |
| Pubblicazione: |
2011
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