Related Experiment Video
Updated: Jun 6, 2026

Implementation of In Vitro Drug Resistance Assays: Maximizing the Potential for Uncovering Clinically Relevant Resistance Mechanisms
Published on: December 9, 2015
RNA interference in the clinic: challenges and future directions
Chad V Pecot1, George A Calin, Robert L Coleman
1U.T. M.D. Anderson Cancer Center, Houston, Texas 77030, USA.
Abstract:
Inherent difficulties with blocking many desirable targets using conventional approaches have prompted many to consider using RNA interference (RNAi) as a therapeutic approach. Although exploitation of RNAi has immense potential as a cancer therapeutic, many physiological obstacles stand in the way of successful and efficient delivery. This Review explores current challenges to the development of synthetic RNAi-based therapies and considers new approaches to circumvent biological barriers, to avoid intolerable side effects and to achieve controlled and sustained release.
Insights
RNA interference (RNAi) offers therapeutic potential, but delivery challenges hinder its use in cancer. This review examines obstacles and new strategies for effective RNAi-based cancer therapies.
Area of Science:
- Biochemistry
- Molecular Biology
- Pharmacology
Background:
- Conventional therapeutic approaches face challenges in targeting specific molecules.
- RNA interference (RNAi) presents a promising alternative for therapeutic intervention.
- Significant physiological barriers impede the effective delivery of RNAi-based therapeutics.
Purpose of the Study:
- To review the current challenges in developing synthetic RNAi-based therapies.
- To explore novel strategies for overcoming biological barriers in RNAi delivery.
- To discuss methods for achieving controlled and sustained release of RNAi agents.
Main Methods:
- Literature review of current research on RNAi therapeutics.
- Analysis of physiological obstacles to RNAi delivery.
- Exploration of innovative approaches to enhance RNAi efficacy and safety.
Main Results:
- Identification of key challenges in synthetic RNAi therapy development.
- Overview of strategies to circumvent biological barriers.
- Discussion of methods for controlled and sustained release of RNAi agents.
Conclusions:
- Overcoming delivery challenges is crucial for realizing the therapeutic potential of RNAi.
- New approaches are needed to improve the safety and efficacy of RNAi-based treatments.
- Further research into delivery systems and controlled release is essential for clinical translation.
Related Concept Videos
RNA Interference
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
Microorganisms in Medicine and Therapeutics
Crossing Over
Homologous Recombination
