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09:09
Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery
Published on: May 2, 2019
Therapeutic siRNA: principles, challenges, and strategies
Kseniya Gavrilov1, W Mark Saltzman
1Department of Cellular and Molecular Physiology, Yale University, New Haven, CT 06520, USA. kseniya.gavrilov@yale.edu
The Yale Journal of Biology and Medicine
|June 28, 2012
Summary
RNA interference (RNAi) offers targeted gene silencing for diseases like cancer. Overcoming in vivo delivery challenges for small interfering RNAs (siRNAs) is key to unlocking RNAi therapeutics potential.
Area of Science:
- Biotechnology
- Molecular Biology
- Genetics
Background:
- RNA interference (RNAi) is a natural gene regulation process.
- RNAi enables sequence-specific gene silencing.
- RNAi has potential for targeted therapeutics in viral diseases and cancer.
Purpose of the Study:
- Review the mechanistic principles of RNA interference.
- Discuss the potential of RNAi therapeutics.
- Identify challenges and engineering solutions for in vivo siRNA delivery.
Main Methods:
- Review of RNA interference mechanisms.
- Analysis of challenges in siRNA delivery.
- Exploration of engineered delivery systems.
Main Results:
- RNAi is a powerful gene silencing tool.
- In vivo delivery of small interfering RNAs (siRNAs) is a major hurdle.
- Delivery systems must improve siRNA stability, targeting, and immune response.
Conclusions:
- RNAi therapeutics hold significant promise.
- Effective siRNA delivery is critical for clinical translation.
- Engineering delivery systems is essential to overcome current limitations.
Keywords:
RNA interferencechemical modificationdeliveryliposomenanoparticlesiRNAtargetingtherapeuticsMore Related Videos
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