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

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...

Full description

Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: Elsevier 2013-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense oligonucleotides
myotonic dystrophy
RNA processing
RNA silencing
triplet repeat expansion
Online Access:http://www.sciencedirect.com/science/article/pii/S216225311630141X

Internet

http://www.sciencedirect.com/science/article/pii/S216225311630141X

Similar Items