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Small interference (RNAi) technique: Exploring its clinical applications, benefits and limitations
Moqbel Ali Moqbel Redhwan1,2, Hariprasad M G1,2, Suman Samaddar3
1Department of Pharmacology, KLE College of Pharmacy, Bengaluru, India.
European Journal of Clinical Investigation
|June 13, 2023
Summary
Small interference RNA (siRNA) shows promise in treating cancers and viral diseases, with 55 clinical trials demonstrating safety and effectiveness. Challenges remain in cellular uptake and targeting for broader therapeutic applications.
Area of Science:
- Biotechnology
- Molecular Biology
- Genetics
Background:
- Small interference RNA (siRNA) is a key technology for gene silencing in research and therapeutics.
- Extensive development has positioned siRNA as a promising therapeutic agent.
- Recent advancements focus on clinical applications and understanding siRNA's potential.
Purpose of the Study:
- To review clinical trials on siRNA over the past five years.
- To analyze the benefits, pharmacokinetics, and safety of siRNA therapeutics.
- To understand the current landscape of siRNA in clinical practice.
Main Methods:
- PubMed database search for 'siRNA' and 'in vivo' clinical trials published within the last five years.
- Analysis of clinical trial features registered on clinicaltrials.gov.
- Inclusion of English-language articles.
Main Results:
- 55 clinical studies on siRNA have been published.
- siRNA demonstrates tolerability, safety, and effectiveness in treating cancers and hereditary/viral diseases.
- Multiple administration routes are available, enabling simultaneous gene silencing, but challenges in cellular uptake, targeting, and elimination persist.
Conclusions:
- siRNA (RNAi) is poised to be a critical technique for combating diverse diseases.
- Despite advantages, clinical applications of RNAi face limitations.
- Overcoming current challenges is crucial for the widespread adoption of siRNA therapies.
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