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Accepted (AM)Open Access (License Unspecified), Open
Journal article
Polymersome delivery of siRNA and antisense oligonucleotides
Journal of controlled release, v 134(2)
12 Nov 2008
PMID: 19084037
Featured in Collection : UN Sustainable Development Goals @ Drexel
Abstract
siRNA and antisense oligonucleotides, AON, have similar size and negative charge and are often packaged for
in vitro
delivery with cationic lipids or polymers–but exposed positive charge is problematic
in vivo
. Here we demonstrate loading and functional delivery of RNAi and AON with non-ionic, nano-transforming polymersomes. These degradable carriers are taken up passively by cultured cells after which the vesicles transform into micelles that allow endolysosomal escape and delivery of either siRNA into cytosol for mRNA knockdown or else AON into the nucleus for exon skipping within pre-mRNA. Polymersome-mediated knockdown appears as efficient as common cationic-lipid transfection and about half as effective as Lentivirus after sustained selection. For AON, initial results also show that intramuscular injection into a mouse model of muscular dystrophy leads to the expected protein expression, which occurs along the entire length of muscle. The lack of cationic groups in antisense polymersomes together with initial tests of efficacy suggests broader utility of these non-viral carriers.
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Details
- Title
- Polymersome delivery of siRNA and antisense oligonucleotides
- Creators
- Younghoon Kim - University of PennsylvaniaManorama Tewari - University of PennsylvaniaJ. David Pajerowski - University of PennsylvaniaShenshen Cai - University of PennsylvaniaShamik Sen - University of PennsylvaniaJason Williams - Drexel UniversityShashank Sirsi - Drexel UniversityGordon Lutz - Drexel UniversityDennis E. Discher - University of Pennsylvania
- Publication Details
- Journal of controlled release, v 134(2)
- Publisher
- Elsevier
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Biochemistry and Molecular Biology
- Web of Science ID
- WOS:000264503900010
- Scopus ID
- 2-s2.0-60649114699
- Other Identifier
- 991019168180304721
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- Collaboration types
- Domestic collaboration
- Web of Science research areas
- Chemistry, Multidisciplinary
- Pharmacology & Pharmacy