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Updated: Jul 7, 2026
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Double-stranded RNA Oral Delivery Methods to Induce RNA Interference in Phloem and Plant-sap-feeding Hemipteran Insects
Published on: May 4, 2018
Delivery vehicles for small interfering RNA in vivo
1Alnylam Pharmaceuticals, Cambridge, MA 02142, USA. tdefougerolles@alnylam.com
Human Gene Therapy
|February 9, 2008
Summary
RNA interference (RNAi) therapeutics, utilizing small interfering RNA (siRNA), offer potent gene silencing for drug development. Overcoming delivery challenges is key to advancing these revolutionary therapies into wider clinical use.
Area of Science:
- Molecular Biology
- Genetics
- Pharmacology
Background:
- RNA interference (RNAi) is a biological mechanism for selective messenger RNA (mRNA) silencing.
- The completion of the human genome sequence enables RNAi-based drug development for various diseases.
- Synthetic small interfering RNA (siRNA) shows potential as a therapeutic agent due to its potent and reversible gene silencing capabilities in vivo.
Purpose of the Study:
- To review advancements in in vivo siRNA delivery technologies for therapeutic applications.
- To discuss the challenges and successes of different siRNA delivery strategies.
- To provide an update on ongoing clinical trials utilizing siRNA therapeutics.
Main Methods:
- Review of existing literature on in vivo siRNA delivery methods.
- Analysis of various delivery approaches including direct administration, nanoparticles, and conjugation techniques.
- Compilation of data on the status of current clinical trials involving siRNA.
Main Results:
- Multiple siRNA delivery approaches have shown success in vivo.
- Direct local administration, liposome- and polymer-based nanoparticles, and conjugation/complexation are effective strategies.
- Five clinical trials are currently active, with more anticipated.
Conclusions:
- Delivery remains a significant hurdle for the broader development of siRNA therapeutics.
- A combination of distinct delivery technologies will likely be necessary, tailored to specific clinical indications, administration routes, and target cells.
- siRNA therapeutics have the potential to become a revolutionary class of drugs.
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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.
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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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Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
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...
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...
02:30Small interfering RNAs (siRNA)
Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
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...
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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PIWI-interacting RNAs, or piRNAs, are the most abundant short non-coding RNAs. More than 20,000 genes have been found in humans that code for piRNAs while only 2000 genes have been found for miRNAs. piRNAs can act at the transcriptional and post-transcriptional levels and have a vital role in silencing transposable elements present in germ cells. They are also involved in epigenetic silencing and activation. Previously, they were thought to function only in germ cells but new evidence suggests...