Related Experiment Video
Updated: Jul 12, 2026
07:56
Bacterial Delivery of RNAi Effectors: Transkingdom RNAi
Published on: August 18, 2010
Development of new RNAi therapeutics
1Immusol, Inc., San Diego, CA 92121, USA.
Histology and Histopathology
|December 7, 2006
Summary
RNA interference (RNAi) therapeutics offer a promising new avenue for drug discovery by enabling gene inactivation. Innovations in delivery and design are overcoming challenges for effective RNAi-based treatments.
Area of Science:
- Biotechnology
- Pharmacology
- Molecular Biology
Background:
- RNA interference (RNAi) is crucial for gene function studies, underpinning drug discovery.
- RNAi-based drugs present an alternative therapeutic approach, potentially accelerating development.
- Significant technological and biological hurdles impede the clinical translation of RNAi therapeutics.
Purpose of the Study:
- To review the progress of RNAi therapeutics in drug development.
- To discuss innovations addressing pharmacokinetic and biological limitations.
- To highlight applications in preclinical models and human trials.
Main Methods:
- Review of recent advancements in nucleic acid chemistry, formulations, and delivery systems.
- Analysis of strategies to mitigate off-target effects, interferon response, and miRNA interference.
- Examination of preclinical data and clinical trial outcomes for local diseases.
Main Results:
- Innovations in chemistry, formulation, and delivery are enabling effective RNAi therapeutics.
- Careful design, informed by RNAi/miRNA biology, can minimize tissue toxicity.
- Favorable results have been observed in human trials for local diseases.
Conclusions:
- RNAi therapeutics hold the potential to revolutionize drug development if systemic application is achieved.
- Overcoming limitations like poor pharmacokinetics and biological restrictions is key to success.
- Continued research and innovation are vital for realizing the full potential of RNAi-based treatments.
Related Concept Videos
01:23Types of RNA
Overview
Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in the regulation of gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
RNA...
Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in the regulation of gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
RNA...
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...
02:30siRNA - 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...
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...
01:20Types of RNA
Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in regulating gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
RNA Performs Diverse...
RNA Performs Diverse...