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

• Main Authors: Anchel González-Barriga (Author), Susan AM Mulders (Author), Jeroen van de Giessen (Author), Jeroen D Hooijer (Author), Suzanne Bijl (Author), Ingeborg DG van Kessel (Author), Josee van Beers (Author), Judith CT van Deutekom (Author), Jack AM Fransen (Author), Bé Wieringa (Author), Derick G Wansink (Author)
• Format: Book
• Published: Elsevier, 2013-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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'-...