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...

Popoln opis

Bibliografske podrobnosti

Principais autores:	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:	Artigo
Jezik:	Inglês
Izdano:	Elsevier 2013-01-01
Serija:	Molecular Therapy: Nucleic Acids
Teme:	antisense oligonucleotides myotonic dystrophy RNA processing RNA silencing triplet repeat expansion
Online dostop:	http://www.sciencedirect.com/science/article/pii/S216225311630141X
Oznake:	Označite Brez oznak, prvi označite!