Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development
University of Connecticut · Yale University · +3 more institutions
Abstract
Antisense oligonucleotides (ASOs) bind sequence specifically to the target RNA and modulate protein expression through several different mechanisms. The ASO field is an emerging area of drug development that targets the disease source at the RNA level and offers a promising alternative to therapies targeting downstream processes. To translate ASO-based therapies into a clinical success, it is crucial to overcome the challenges associated with off-target side effects and insufficient biological activity. In this regard, several chemical modifications and diverse delivery strategies have been explored. In this review, we systematically discuss the chemical modifications, mechanism of action, and optimized…
Citation impact
- FWCI
- 19.42
- Percentile
- 100%
- References
- 138
Authors
7Topics & keywords
- Medicine
- Drug development
- Drug discovery
- Food and drug administration
- Drug
- Oligonucleotide
- Computational biology
- Site of action