The DOI matches its Crossref record; 2 of the 2 references it cites were found in a registry or an index.
Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA
Open accessAbstract
Resisting Repeats: A set of diseases, including myotonic dystrophy, are caused by the expansion of a simple repeat in genomic DNA, which, when transcribed into RNA, can be toxic to other cellular processes. Ameliorating the effects of this toxic, repeat-laden RNA may also relieve the symptoms of the disease. Wheeler et al. (p. 336 ; see the Perspective by Cooper ) developed an antisense morpholino oligonucleotide complementary to the expanded repeats found in the myotonic dystrophy protein kinase messenger RNA (mRNA). The morpholino bound the repeats in vitro and displaced the inappropriately bound and sequestered RNA splicing factor, Muscleblind-like 1. In an in vivo mouse model for myotonic dystrophy, local injection of the morpholino corrected a number of cellular defects in muscle, including the alternative mRNA splicing of several genes, among them the muscle-specific chloride channel, CIC1, leading to a marked reduction in the myotonia.
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Citations
Cites
Failure of MBNL1-dependent post-natal splicing transitions in myotonic dystrophy
Lin, X., Miller, J. W., Mankodi, A., Kanadia, R. N., Yuan, Y., Moxley, R. T., Swanson, M. S., & Thornton, C. A. (2006) · Human Molecular Genetics · doi:10.1093/hmg/ddl132 · Cited by 510
Self-citationRNA toxicity is a component of ataxin-3 degeneration in Drosophila
Li, L.-B., Yu, Z., Teng, X., & Bonini, N. M. (2008) · Nature · doi:10.1038/nature06909 · Cited by 316
Cited by
Bennett, C. F., & Swayze, E. E. (2010) · Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev.pharmtox.010909.105654 · Cited by 1,374
RNA therapeutics: beyond RNA interference and antisense oligonucleotides
Kole, R., Krainer, A. R., & Altman, S. (2012) · Nature Reviews Drug Discovery · doi:10.1038/nrd3625 · Cited by 1,152
Scotti, M. M., & Swanson, M. S. (2016) · Nature Reviews Genetics · doi:10.1038/nrg.2015.3 · Cited by 1,163
RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention
Donnelly, C. J., Zhang, P.-W., Pham, J. T., Haeusler, A. R., Mistry, N. A., Vidensky, S., Daley, E. L., Poth, E. M., Hoover, B., Fines, D. M., et al. (2013) · Neuron · doi:10.1016/j.neuron.2013.10.015 · Cited by 877
Antisense oligonucleotides: the next frontier for treatment of neurological disorders
Rinaldi, C., & Wood, M. J. A. (2018) · Nature Reviews Neurology · doi:10.1038/nrneurol.2017.148 · Cited by 788
Metadata
- Authors
- Wheeler, T. M., Sobczak, K., Lueck, J. D., Osborne, R. J., Lin, X., Dirksen, R. T., & Thornton, C. A.
- Venue
- Science
- Published
- 2009-07-17
- Volume
- 325
- Issue
- 5938
- Pages
- 336-339
- ISSN
- 0036-8075 (ISSN-L)
- 1095-9203
- OpenAlex
- W2084091637
- Cited by
- 387
- References
- 2
- FWCI
- 15.68
- Type
- journal article
- Language
- en
- Open access
- green
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- CoChat. (2026). Citation check: Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA. https://app.cochat.ai/papers/10.1126/science.1173110
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- Wheeler, T. M., Sobczak, K., Lueck, J. D., Osborne, R. J., Lin, X., Dirksen, R. T., & Thornton, C. A. (2009). Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA. Science. https://doi.org/10.1126/science.1173110
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