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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.
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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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Experimental RNAi02:15

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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 Editing02:23

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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The Upf proteins that carry out nonsense-mediated decay (NMD) are found in all eukaryotic organisms, including humans. Each protein has an individual role, but they need to work in collaboration. Upf1 is an ATP-dependent RNA helicase that unwinds the RNA helix. Because Upf1 can unwind any RNA, Upf2 and Upf3 are required to help Upf1 discriminate between nonsense and normal mRNAs.
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Updated: Nov 17, 2025

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Antisense technology: A review.

Stanley T Crooke1, Xue-Hai Liang1, Brenda F Baker2

  • 1Core Antisense Research, Ionis Pharmaceuticals, Inc, Carlsbad, California, USA.

The Journal of Biological Chemistry
|February 18, 2021
PubMed
Summary
This summary is machine-generated.

Antisense technology, including single-strand antisense drugs (ASOs) and double-strand ASOs (siRNAs), is advancing rapidly with approved therapies and promising clinical trials for various diseases. This review highlights molecular mechanisms and clinical outcomes, offering a prospective view of this innovative RNA-targeting field.

Keywords:
RNase H1antisenseclinical resultsmolecular mechanismssplicing

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Pharmacology

Background:

  • Antisense technology has matured, with ten RNA-targeted drugs (eight single-strand antisense drugs (ASOs) and two double-strand ASOs (siRNAs)) now approved.
  • ASOs in clinical trials exhibit innovation in delivery methods and target a spectrum of rare and common diseases, including cardiovascular conditions.

Purpose of the Study:

  • To review the molecular mechanisms underlying the efficacy of antisense technology.
  • To exemplify specific molecular mechanisms and challenges using recent clinical results of various ASOs.
  • To provide a prospective outlook on the future of antisense technology.

Main Methods:

  • Focus on molecular events driving observed effects of antisense drugs.
  • Analysis of recent clinical trial data involving multiple ASOs.
  • Literature review of advancements and applications in antisense technology.

Main Results:

  • Ten RNA-targeted drugs, including ASOs and siRNAs, are commercially available.
  • ASOs are being investigated in large-scale cardiovascular outcome studies and other significant clinical trials.
  • Innovative delivery routes and diverse disease targets characterize current ASO development.

Conclusions:

  • Antisense technology is demonstrating significant clinical success and therapeutic potential.
  • Understanding molecular mechanisms is crucial for optimizing ASO therapies.
  • The field is poised for continued growth and broader application in medicine.