The DOI matches its Crossref record.
Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing
Abstract
An estimated 60% of all human genes undergo alternative splicing, a highly regulated process that produces splice variants with different functions. Such variants have been linked to a variety of cancers, and genetic diseases such as thalassemia and cystic fibrosis. This Perspective describes a promising approach to RNA repair based on the use of antisense oligonucleotides to modulate alternative splicing and engender the production of therapeutic gene products.
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Citations
Cited by
Cooper, T. A., Wan, L., & Dreyfuss, G. (2009) · Cell · doi:10.1016/j.cell.2009.02.011 · Cited by 1,159
Targeting RNA structures with small molecules
Childs-Disney, J. L., Yang, X., Gibaut, Q. M. R., Tong, Y., Batey, R. T., & Disney, M. D. (2022) · Nature Reviews Drug Discovery · doi:10.1038/s41573-022-00521-4 · Cited by 587
Scaffidi, P., & Misteli, T. (2005) · Nature Medicine · doi:10.1038/nm1204 · Cited by 553
Alternative splicing in disease and therapy
Garcia-Blanco, M. A., Baraniak, A. P., & Lasda, E. L. (2004) · Nature Biotechnology · doi:10.1038/nbt964 · Cited by 527
Human laminopathies: nuclei gone genetically awry
Capell, B. C., & Collins, F. S. (2006) · Nature Reviews Genetics · doi:10.1038/nrg1906 · Cited by 527
Metadata
- Authors
- Sazani, P., & Kole, R.
- Venue
- Journal of Clinical Investigation
- Published
- 2003-08-15
- Volume
- 112
- Issue
- 4
- Pages
- 481-486
- ISSN
- 0021-9738 (ISSN-L)
- OpenAlex
- W4252995669
- Cited by
- 198
- References
- 0
- FWCI
- 4.93
- Type
- journal article
- Language
- en
- Open access
- closed
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- CoChat. (2026). Citation check: Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing. https://app.cochat.ai/papers/10.1172/jci200319547
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- Sazani, P., & Kole, R. (2003). Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing. Journal of Clinical Investigation. https://doi.org/10.1172/jci200319547
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
