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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Bibliografski detalji
Izdano u:Nature
Glavni autori: Wheeler, Thurman M., Leger, Andrew J., Pandey, Sanjay K., MacLeod, A. Robert, Nakamori, Masayuki, Cheng, Seng H., Wentworth, Bruce M., Bennett, C. Frank, Thornton, Charles A.
Format: Artigo
Jezik:Inglês
Izdano: 2012
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Article
Online pristup: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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