Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular Dystrophy
Open Access
- 1 June 2012
- journal article
- Published by Elsevier BV in Molecular Therapy
- Vol. 20 (6), 1212-1221
- https://doi.org/10.1038/mt.2012.26
Abstract
No abstract availableKeywords
This publication has 55 references indexed in Scilit:
- Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation studyThe Lancet, 2011
- Sustained alpha‐sarcoglycan gene expression after gene transfer in limb‐girdle muscular dystrophy, type 2DAnnals of Neurology, 2010
- Exon‐skipped dystrophins for treatment of Duchenne muscular dystrophy: Mass spectrometry mapping of most exons and cooperative domain designs based on single molecule mechanicsCytoskeleton, 2010
- Immune Responses to AAV in Canine Muscle Monitored by Cellular Assays and Noninvasive ImagingMolecular Therapy, 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
- Enhanced Exon-skipping Induced by U7 snRNA Carrying a Splicing Silencer Sequence: Promising Tool for DMD TherapyMolecular Therapy, 2009
- Differential stabilities of alternative exon-skipped rod motifs of dystrophinBiochimica et Biophysica Acta (BBA) - Proteins and Proteomics, 2009
- A Single Intravenous Injection of Adeno-associated Virus Serotype-9 Leads to Whole Body Skeletal Muscle Transduction in DogsMolecular Therapy, 2008
- Antisense Oligonucleotide-induced Exon Skipping Across the Human Dystrophin Gene TranscriptMolecular Therapy, 2007
- Targeted Exon Skipping in Transgenic hDMD Mice: A Model for Direct Preclinical Screening of Human-Specific Antisense OligonucleotidesMolecular Therapy, 2004