Small molecule modifiers of the microRNA and RNA interference pathway
1Department of Chemistry, North Carolina State University, Raleigh, North Carolina 27695-8204, USA. alex_deiters@ncsu.edu
The AAPS Journal
|November 26, 2009
Summary
Small molecules can now target the RNA interference (RNAi) pathway, offering new ways to control gene silencing. This approach holds promise for developing novel cancer therapeutics by modulating microRNAs (miRNAs).
Area of Science:
- Molecular Biology
- Genetics
- Biochemistry
Background:
- The RNA interference (RNAi) pathway is a natural process for gene silencing using small double-stranded RNA molecules.
- MicroRNAs (miRNAs), processed via the RNAi pathway, are endogenous regulators of gene expression.
- Dysregulation of miRNAs is implicated in various human diseases, particularly cancer.
Purpose of the Study:
- To explore the therapeutic potential of chemically modulating the RNA interference (RNAi) pathway.
- To investigate the use of small molecules for activating tumor-suppressive miRNAs and inhibiting oncogenic miRNAs.
Main Methods:
- Focus on the chemical modulation of RNAi pathway components.
- Utilizing small molecule inhibitors and activators targeting RNAi and miRNA processes.
Main Results:
- Small molecule targeting of RNAi is a feasible approach.
- Chemical modulation of miRNAs can influence disease states, including cancer.
Conclusions:
- Small molecules offer a novel therapeutic strategy for diseases linked to miRNA dysregulation.
- Targeting RNAi and miRNAs with small molecules presents a promising avenue for cancer drug development.
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...
01:22MicroRNAs
MicroRNA (miRNA) are short, regulatory RNA transcribed from introns (non-coding regions of a gene) or intergenic regions (stretches of DNA present between genes). Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself, forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA...
01:22MicroRNAs
MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA ends...
01:22MicroRNAs
MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA ends...
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...