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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...

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Hoofdauteurs: Anchel González-Barriga, Susan AM Mulders, Jeroen van de Giessen, Jeroen D Hooijer, Suzanne Bijl, Ingeborg DG van Kessel, Josee van Beers, Judith CT van Deutekom, Jack AM Fransen, Bé Wieringa, Derick G Wansink
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Elsevier 2013-01-01
Reeks:Molecular Therapy: Nucleic Acids
Onderwerpen:
antisense oligonucleotides
myotonic dystrophy
RNA processing
RNA silencing
triplet repeat expansion
Online toegang:http://www.sciencedirect.com/science/article/pii/S216225311630141X
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