Effective Exon Skipping and Restoration of Dystrophin Expression by Peptide Nucleic Acid Antisense Oligonucleotides in mdx Mice
Open Access
- 1 January 2008
- journal article
- Published by Elsevier in Molecular Therapy
- Vol. 16 (1) , 38-45
- https://doi.org/10.1038/sj.mt.6300329
Abstract
No abstract availableKeywords
This publication has 27 references indexed in Scilit:
- Peptide-based delivery of nucleic acids: design, mechanism of uptake and applications to splice-correcting oligonucleotidesBiochemical Society Transactions, 2007
- RNA targeting with peptide conjugates of oligonucleotides, siRNA and PNABlood Cells, Molecules, and Diseases, 2006
- Induction of Dystrophin Expression by Exon Skipping in mdx Mice Following Intramuscular Injection of Antisense Oligonucleotides Complexed with PEG–PEI CopolymersMolecular Therapy, 2006
- Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathologyNature Medicine, 2006
- Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heartNature Biotechnology, 2005
- Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cellsGene Therapy, 2004
- Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophyThe Journal of Gene Medicine, 2002
- Antisense Oligonucleotides: Promise and RealityAnnual Review of Pharmacology and Toxicology, 2001
- In Vivo Protein Transduction: Delivery of a Biologically Active Protein into the MouseScience, 1999
- Subcellular fractionation of dystrophin to the triads of skeletal muscleNature, 1987