Related Experiment Video
Updated: Jul 7, 2026
12:03
Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
Published on: September 5, 2016
Review article: RNA interference--potential therapeutic applications for the gastroenterologist
R S Pellish1, A Nasir, B Ramratnam
1Division of Gastroenterology, Warren Alpert Medical School of Brown University, Rhode Island Hospital, Providence, RI 02903, USA.
Alimentary Pharmacology & Therapeutics
|February 6, 2008
Summary
RNA interference (RNAi) offers a new way to control gene expression for treating gastrointestinal and liver diseases. Further research into delivery and side effects is needed for clinical use.
Area of Science:
- Molecular Biology
- Genetics
- Gastroenterology
Background:
- RNA interference (RNAi) is a gene regulation technique using short double-stranded RNA.
- RNAi selectively inhibits gene expression post-transcriptionally.
- Gastrointestinal and hepatic disorders are promising targets for RNAi therapy due to drug delivery advantages.
Purpose of the Study:
- To review literature on RNAi applications in gastroenterology.
- To explore future RNAi-based therapies for luminal diseases.
Main Methods:
- Literature search using PubMed with the term 'RNA interference'.
- Focus on studies related to therapeutic uses in gastrointestinal and liver diseases.
Main Results:
- Cellular and animal models show potential for short-interfering RNA (siRNA) in treating viral hepatitis and inflammatory bowel disease.
- siRNA-based therapies may offer a new frontier in molecular-targeted treatments for gastroenterology and hepatology.
Conclusions:
- Short-interfering RNA (siRNA) enables specific gene expression inhibition.
- Clinical translation requires further investigation of side effects and delivery systems.
- Mucosal or endoscopic delivery of siRNA may lead to early clinical applications.
Related Concept Videos
01:23RNA Interference
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...
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...
01:23RNA Interference
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...
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...
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...
02:30siRNA - Small Interfering RNAs
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...
01:30Inhibitors of Viral Protein Synthesis
Protein synthesis is indispensable for viral replication, as viruses lack the cellular machinery required for this process and must hijack the host's translational apparatus. In response, host cells deploy a critical innate immune defense involving interferons, specialized cytokines that play a central role in inhibiting viral propagation.Upon viral detection, infected cells release interferons that bind to receptors on adjacent uninfected cells, activating the JAK-STAT signaling pathway and...
01:29Microorganisms in Medicine and Therapeutics
Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.