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Related Concept Videos

Experimental RNAi02:15

Experimental 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...
RNA Interference01:23

RNA 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.
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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...
Small interfering RNAs (siRNA)02:30

Small interfering RNAs (siRNA)

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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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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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Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells
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Engineering small interfering RNAs by strategic chemical modification.

Jesper B Bramsen1, Jørgen Kjems

  • 1Department of Molecular Biology and Genetics, Interdisciplinary Nanoscience Center (iNANO), University of Aarhus, Aarhus, Denmark. jebb@mb.au.dk

Methods in Molecular Biology (Clifton, N.J.)
|October 3, 2012
PubMed
Summary

Chemical modifications enhance synthetic small interfering RNAs (siRNAs) for improved in vitro and in vivo applications, addressing delivery and off-target challenges in functional genomics and therapeutics.

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Area of Science:

  • Molecular Biology
  • RNA Therapeutics
  • Functional Genomics

Background:

  • Synthetic small interfering RNAs (siRNAs) are powerful tools for functional genomics in cell cultures.
  • siRNA applications face challenges in vivo, including delivery, off-target effects, and immunogenicity.
  • Current limitations hinder the full potential of siRNAs in therapeutics.

Purpose of the Study:

  • To review literature on chemical modifications of siRNAs.
  • To provide a guide for improving siRNA performance through chemical engineering.
  • To address challenges in siRNA delivery, off-target effects, and immunogenicity.

Main Methods:

  • Literature review of chemical modifications in siRNA synthesis.
  • Analysis of nucleotide modifications for enhanced siRNA properties.
  • Guidance on tailoring modifications for specific in vitro and in vivo applications.

Main Results:

  • Chemical modifications can significantly improve siRNA reliability and efficiency.
  • Strategic incorporation of modifications addresses in vivo delivery and off-target concerns.
  • Modified siRNAs show potential for overcoming immunogenicity issues.

Conclusions:

  • Chemical engineering of siRNAs is crucial for advancing their use in research and therapy.
  • Optimized siRNA modifications can unlock broader applications in functional genomics and medicine.
  • This review offers practical insights into leveraging siRNA chemical modifications effectively.