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Targeting nuclear RNA for in vivo correction of myotonic dystrophy
Antisense oligonucleotides (ASOs) hold promise for gene-specific knockdown in diseases that involve RNA or protein gain-of-function. In the hereditary degenerative disease myotonic dystrophy type 1 (DM1), transcripts from the mutant allele contain an expanded CUG repeat(1–3) and are retained in the...
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| Publicado no: | Nature |
|---|---|
| Principais autores: | , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2012
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4221572/ https://ncbi.nlm.nih.gov/pubmed/22859208 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nature11362 |
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