Cationic PMMA Nanoparticles Bind and Deliver Antisense Oligoribonucleotides Allowing Restoration of Dystrophin Expression in the mdx Mouse
Open Access
- 1 May 2009
- journal article
- Published by Elsevier in Molecular Therapy
- Vol. 17 (5) , 820-827
- https://doi.org/10.1038/mt.2009.8
Abstract
No abstract availableThis publication has 35 references indexed in Scilit:
- Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and functionHuman Molecular Genetics, 2008
- Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx MiceMolecular Therapy, 2008
- Functionalized PEG–PEI Copolymers Complexed to Exon-Skipping Oligonucleotides Improve Dystrophin Expression inmdxMiceHuman Gene Therapy, 2008
- Nanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of mdx miceBMC Biotechnology, 2008
- Polymers for intracellular delivery of nucleic acidsJournal of Materials Chemistry, 2007
- Antisense-mediated exon skipping: A versatile tool with therapeutic and research applicationsRNA, 2007
- Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMDGene Therapy, 2006
- Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathologyNature Medicine, 2006
- Targeted Exon Skipping in Transgenic hDMD Mice: A Model for Direct Preclinical Screening of Human-Specific Antisense OligonucleotidesMolecular Therapy, 2004
- Lactosylated polyethylenimine for gene transfer into airway epithelial cells: role of the sugar moiety in cell delivery and intracellular trafficking of the complexesThe Journal of Gene Medicine, 2004