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Published on: August 9, 2019
Development of RNA aptamer-based therapeutic agents
1Department of Chemistry, University of South Florida, 4202 E. Fowler Ave, Tampa, FL 33620, USA.
Current Medicinal Chemistry
|June 11, 2013
Summary
RNA aptamers, small non-coding RNAs, show high specificity for targets. These molecules are promising therapeutic agents for diseases and drug delivery, with several examples in clinical trials.
Area of Science:
- Biochemistry
- Molecular Biology
- Pharmacology
Background:
- RNA aptamers are small, non-coding RNA molecules.
- They exhibit high specificity and affinity for target molecules.
- RNA aptamers function similarly to antibodies in biological systems.
Purpose of the Study:
- To summarize recent advancements in RNA aptamer therapeutics.
- To highlight promising RNA aptamer candidates in clinical and pre-clinical development.
- To discuss the potential of RNA aptamers in disease treatment and targeted drug delivery.
Main Methods:
- In vitro selection using SELEX (Systemic Evolution of Ligands by Exponential Enrichment).
- Review of current clinical trial and pre-clinical data for RNA aptamer therapeutics.
- Analysis of RNA aptamer mechanisms for inhibiting, modulating, and disrupting target proteins.
Main Results:
- RNA aptamers can effectively inhibit, modulate, and disrupt target protein functions.
- Several RNA aptamer-based therapeutics are progressing through clinical trials.
- Pioneer RNA aptamer examples demonstrate therapeutic potential in pre-clinical studies.
Conclusions:
- RNA aptamers represent a significant advancement in therapeutic development.
- Their high specificity and affinity make them suitable for targeted drug delivery.
- Further development of RNA aptamers holds promise for treating various diseases.
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