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Related Concept Videos

RNA Interference01:23

RNA Interference

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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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RNA Interference01:23

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siRNA - Small Interfering RNAs02:30

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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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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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Types of RNA01:20

Types of RNA

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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.
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Types of RNA01:23

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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.
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Cell Based Assays of SINEUP Non-coding RNAs That Can Specifically Enhance mRNA Translation
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siRNA and RNAi optimization.

Adele Alagia1, Ramon Eritja1

  • 1Chemical and Biomolecular Nanotechnology, CIBER-BBN, Institute for Advanced Chemistry of Catalonia, IQAC-CSIC, Barcelona, Spain.

Wiley Interdisciplinary Reviews. RNA
|February 4, 2016
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Summary
This summary is machine-generated.

RNA interference (RNAi) offers therapeutic potential for diseases by downregulating specific genes. Advances in small interfering RNA (siRNA) molecule optimization and delivery systems are overcoming challenges for clinical applications.

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • RNA interference (RNAi) is a posttranscriptional gene regulatory mechanism.
  • Small interfering RNA (siRNA) has significant clinical potential for treating diseases.
  • Over 50 RNA-based drugs are currently in clinical trials.

Purpose of the Study:

  • To review recent literature on optimizing siRNA molecules for therapeutic use.
  • To focus on advances in improving siRNA pharmacokinetic properties.
  • To analyze the impact of RNA modifications on off-target effects and delivery.

Main Methods:

  • Literature review of recent advances in siRNA optimization.
  • Analysis of siRNA molecule properties and their impact on pharmacokinetics.
  • Examination of RNA modifications and their effects on off-target effects (OTEs).
  • Review of novel delivery systems for siRNA therapeutics.

Main Results:

  • RNA modifications can mitigate off-target effects like immunostimulation and miRNA-like effects.
  • Optimized siRNA molecules show improved pharmacokinetic properties.
  • Novel delivery systems enhance siRNA targeting and efficacy.
  • Addressing OTEs and delivery are crucial for therapeutic success.

Conclusions:

  • Modified siRNA molecules and advanced delivery systems represent significant progress.
  • These developments pave the way for reliable siRNA therapeutics.
  • RNAi technology continues to advance towards clinical viability.