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Related Concept Videos

Experimental RNAi02:15

Experimental RNAi

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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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siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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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...
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MicroRNAs01:22

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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...
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MicroRNAs01:22

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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...
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RNA Interference01:23

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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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Tissue-specific gene silencing monitored in circulating RNA.

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    RNA (New York, N.Y.)
    |December 21, 2013
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    Researchers found that tissue-specific messenger RNAs (mRNAs) in bodily fluids can track gene silencing. This discovery aids the clinical development of RNA-based therapeutics by enabling noninvasive monitoring of RNA modulation.

    Keywords:
    RNAicirculating RNAexosomegene silencingin vivo delivery

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    Area of Science:

    • Molecular Biology
    • Pharmacology
    • Biochemistry

    Background:

    • Pharmacologic target gene modulation is key for RNA antagonist strategies and gene therapy.
    • Monitoring RNA modulation in tissues noninvasively is crucial for therapeutic development.

    Purpose of the Study:

    • To detect tissue-specific messenger RNAs (mRNAs) in biological fluids.
    • To establish a noninvasive method for monitoring RNA modulation in tissues.
    • To assess the efficacy of RNA-based therapeutic strategies.

    Main Methods:

    • Detection of tissue-specific mRNAs in serum and cerebrospinal fluid.
    • RNA interference (RNAi)-mediated gene silencing in the liver and brain.
    • Administration of anti-microRNA (miRNA) oligonucleotides.
    • Quantification of ectopic adenoviral transgene expression via serum mRNA levels.

    Main Results:

    • Tissue-specific mRNAs were detected in biological fluids.
    • RNAi-mediated gene silencing led to reduced mRNA levels in serum and cerebrospinal fluid.
    • Anti-miRNA oligonucleotide administration decreased circulating miRNA levels.
    • Serum mRNA measurements accurately quantified transgene expression in the liver.

    Conclusions:

    • Detection of circulating mRNAs provides a noninvasive readout of tissue-specific gene modulation.
    • This method can significantly advance the clinical development of RNA-based therapeutics.
    • Monitoring RNA levels in biological fluids offers a powerful tool for assessing therapeutic efficacy.