Journal article
Nucleobase-modified antisense oligonucleotides containing 5-(phenyltriazol)-2′-deoxyuridine nucleotides induce exon-skipping in vitro
RSC Advances, Vol.7(86), pp.54542-54545
2017
Abstract
Chemically-modified antisense oligonucleotide-mediated exon-skipping has been validated as a therapeutic strategy for tackling several disease pathologies, particularly duchenne muscular dystrophy. To date, only sugar-modified and internucleotide linkage-modified oligonucleotide chemistries have been explored for exon-skipping applications. Herein, for the first time, we have investigated the potential of nucleobase-modified antisense oligonucleotides to induce exon-skipping. For this purpose, we have synthesised 5-(phenyltriazol)-2′-deoxyuridine-modified 2′-O-methyl mixmer antisense oligonucleotides, and evaluated their efficacy to induce exon-23 skipping in H-2Kb-tsA58 (H2K) mdx mouse myotubes as a model system. Our results showed that the phenyltriazol base-modified AOs successfully induced efficient exon-skipping in a DMD transcript. Our findings open up the exploration of novel base-modified antisense oligonucleotides for exon-skipping applications.
Details
- Title
- Nucleobase-modified antisense oligonucleotides containing 5-(phenyltriazol)-2′-deoxyuridine nucleotides induce exon-skipping in vitro
- Authors/Creators
- B.T. Le (Author/Creator) - Murdoch UniversityM. Hornum (Author/Creator) - Department of Physics, Chemistry and PharmacyP.K. Sharma (Author/Creator) - Kurukshetra UniversityP. Nielsen (Author/Creator) - University of Southern DenmarkR.N. Veedu (Author/Creator) - Murdoch University
- Publication Details
- RSC Advances, Vol.7(86), pp.54542-54545
- Publisher
- Royal Society of Chemistry
- Identifiers
- 991005540097707891
- Copyright
- © 2017 Royal Society of Chemistry
- Murdoch Affiliation
- Centre for Comparative Genomics
- Language
- English
- Resource Type
- Journal article
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