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Updated: Jun 23, 2026
09:09
Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery
Published on: May 2, 2019
Overcoming obstacles to develop effective and safe siRNA therapeutics
1siRNA Therapeutics, Abbott Laboratories, Abbott Park, IL 60031, USA.
Expert Opinion on Biological Therapy
|April 28, 2009
Summary
RNA interference (RNAi) therapy shows promise by targeting mRNA, but effective delivery remains a challenge. Advances in delivery systems are crucial for realizing the full therapeutic potential of RNAi treatments.
Area of Science:
- Biotechnology
- Molecular Biology
- Therapeutics
Background:
- RNA interference (RNAi) offers a novel therapeutic approach by targeting messenger RNA (mRNA).
- It bypasses the challenge of targeting disease-causing genes that are difficult to drug.
- The primary obstacle to widespread RNAi therapy is the absence of safe and effective delivery methods.
Purpose of the Study:
- To review the progress in RNAi therapeutics.
- To identify the remaining challenges in developing RNAi therapies.
Main Methods:
- Focuses on the design principles of RNAi delivery technologies.
- Examines systems that have shown target inhibition in vivo following systemic administration.
Main Results:
- Numerous promising delivery technologies have emerged.
- All current delivery platforms exhibit limitations.
Conclusions:
- Optimal combinations of disease, target, and RNA trigger may enable near-term RNAi therapeutic advancements despite imperfect delivery.
- High-throughput assays are essential for evaluating and comparing in vivo delivery system performance for siRNA development.
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In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the ATP-dependent...
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02:15Experimental RNAi
RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
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...
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