Design and Analysis of Effects of Triplet Repeat Oligonucleotides in Cell Models for Myotonic Dystrophy

Myotonic dystrophy type 1 (DM1) is caused by DM protein kinase (DMPK) transcripts containing an expanded (CUG)n repeat. Antisense oligonucleotide (AON)-mediated suppression of these mutant RNAs is considered a promising therapeutic strategy for this severe disorder. Earlier, we identified a 2′-O-met...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: González-Barriga, Anchel, Mulders, Susan AM, van de Giessen, Jeroen, Hooijer, Jeroen D, Bijl, Suzanne, van Kessel, Ingeborg DG, van Beers, Josee, van Deutekom, Judith CT, Fransen, Jack AM, Wieringa, Bé, Wansink, Derick G
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Nature Publishing Group 2013
Pynciau:
Original Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC3615819/
https://ncbi.nlm.nih.gov/pubmed/23511335
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2013.9
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