Journal article
Novel disulfide-bridged bioresponsive antisense oligonucleotide induces efficient splice modulation in muscle Myotubes in Vitro
ACS Omega, Vol.5(29), pp.18035-18039
2020
Abstract
Splice-modulating antisense therapy has shown tremendous potential in therapeutic development in recent years with four FDA-approved antisense drugs since 2016. However, an efficient and nontoxic antisense oligonucleotide (AO) delivery system still remains as a major obstacle in nucleic acid therapeutics field. Vitamin-E (α-tocopherol) is an essential dietary requirement for human body. This fat-soluble compound is one of the most important antioxidants which involves in numerous biological pathways. In this study, for the first time, we explored the scope of using α-tocopherol-conjugated bioresponsive AOs to induce splice modulation in mouse muscle myotubes in vitro. Our results showed that the bioresponsive construct efficiently internalized into the cell nucleus and induced exon 23 skipping in mdx mouse myotubes. Based on our exciting new results, we firmly believe that our findings could potentially benefit toward establishing a delivery approach to advance the field of splice-modulating AO therapy.
Details
- Title
- Novel disulfide-bridged bioresponsive antisense oligonucleotide induces efficient splice modulation in muscle Myotubes in Vitro
- Authors/Creators
- B.T. Le (Author/Creator) - Murdoch UniversityT.R. Kosbar (Author/Creator) - Murdoch UniversityR.N. Veedu (Author/Creator) - Murdoch University
- Publication Details
- ACS Omega, Vol.5(29), pp.18035-18039
- Publisher
- American Chemical Society
- Identifiers
- 991005542735207891
- Copyright
- © 2020 American Chemical Society
- Murdoch Affiliation
- Centre for Molecular Medicine and Innovative Therapeutics
- Language
- English
- Resource Type
- Journal article
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Source: InCites
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- Collaboration types
- Domestic collaboration
- Citation topics
- 2 Chemistry
- 2.170 Nucleic Acids Chemistry
- 2.170.988 Oligonucleotide Modifications
- Web Of Science research areas
- Chemistry, Multidisciplinary
- ESI research areas
- Chemistry