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Related Concept Videos

siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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 ATP-dependent...
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.
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...
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.
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)

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 ATP-dependent...
MicroRNAs01:22

MicroRNAs

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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In Vitro Selection of Engineered Transcriptional Repressors for Targeted Epigenetic Silencing
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Published on: May 5, 2023

Multimeric small interfering ribonucleic acid for highly efficient sequence-specific gene silencing.

Hyejung Mok1, Soo Hyeon Lee, Ji Won Park

  • 1Department of Biological Sciences and Graduate School of Nanoscience and Technology, Korea Advanced Institute of Science and Technology, Daejeon, 305-701, South Korea.

Nature Materials
|January 26, 2010
PubMed
Summary

Chemically modified small interfering RNA (siRNA) forms stable complexes for improved gene silencing. This novel approach enhances therapeutic delivery and efficacy for various diseases.

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Area of Science:

  • Biochemistry
  • Molecular Biology
  • RNA Therapeutics

Background:

  • Small interfering RNA (siRNA) is a therapeutic agent for gene silencing at the post-transcriptional level.
  • Safe and efficient delivery of siRNA therapeutics remains a significant challenge for clinical applications.

Purpose of the Study:

  • To develop a novel biologically active siRNA structure for enhanced gene silencing.
  • To address the delivery and stability issues associated with current siRNA therapeutics.

Main Methods:

  • Chemically self-crosslinked and multimerized siRNA using cleavable disulfide linkages.
  • Formation of polyelectrolyte complexes with cationic carriers.
  • In vitro and in vivo evaluation of gene-silencing efficiencies and immune induction.

Main Results:

  • Multimerized siRNA forms more stable and compact complexes with reduced cytotoxicity compared to naked siRNA.
  • Enhanced gene-silencing efficiencies were observed in vitro and in vivo.
  • The modified siRNA did not significantly elicit immune responses.

Conclusions:

  • The novel multimerized siRNA structure offers a promising platform for developing effective gene-silencing therapeutics.
  • Complexation with cationic condensing agents further enhances the therapeutic potential of this modified siRNA.
  • This approach holds potential for treating various diseases through targeted gene silencing.