In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse
Open Access
- 1 November 2010
- journal article
- Published by Elsevier BV in Molecular Therapy
- Vol. 18 (11), 1995-2005
- https://doi.org/10.1038/mt.2010.186
Abstract
No abstract availableKeywords
This publication has 52 references indexed in Scilit:
- Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse ModelMolecular Therapy, 2010
- In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophyThe Journal of Gene Medicine, 2010
- Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept studyThe Lancet Neurology, 2009
- Rational Design of Antisense Oligomers to Induce Dystrophin Exon SkippingMolecular Therapy, 2009
- Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating MechanismsMolecular Therapy, 2009
- Antisense Oligonucleotide-induced Exon Skipping Across the Human Dystrophin Gene TranscriptMolecular Therapy, 2007
- Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathologyNature Medicine, 2006
- Functional Analysis of 114 Exon-Internal AONs for Targeted DMD Exon Skipping: Indication for Steric Hindrance of SR Protein Binding SitesOligonucleotides, 2005
- Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotideThe Journal of Gene Medicine, 2005
- Targeted Exon Skipping in Transgenic hDMD Mice: A Model for Direct Preclinical Screening of Human-Specific Antisense OligonucleotidesMolecular Therapy, 2004