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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 ends...

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Chitosan/Interfering RNA Nanoparticle Mediated Gene Silencing in Disease Vector Mosquito Larvae
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Gene silencing by chemically modified siRNAs.

Joachim W Engels1

  • 1Goethe-University, Institute of Organic Chemistry and Chemical Biology, Max-von-Laue-Strasse 7, 60438 Frankfurt am Main, Germany. joachim.engels@chemie.uni-frankfurt.de

New Biotechnology
|July 24, 2012
PubMed
Summary

RNA interference (RNAi) offers a promising therapeutic approach by utilizing small interfering RNAs (siRNAs) to target gene function. This review details chemical modifications and design strategies to enhance siRNA therapeutics, addressing immunogenicity and off-target effects.

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Pharmacology

Background:

  • RNA interference (RNAi) is a key tool for gene function validation in research.
  • RNAi therapeutics present a novel paradigm with advantages over traditional drugs.
  • RNAi can target proteins previously inaccessible to drug development.

Purpose of the Study:

  • To review the development of chemically modified small interfering RNAs (siRNAs) over the past decade.
  • To discuss the application of these modified siRNAs as potential therapeutics.
  • To highlight critical aspects of siRNA design, chemical modification, and safety.

Main Methods:

  • Review of literature on chemically modified siRNAs.
  • Analysis of siRNA design principles for therapeutic efficacy.
  • Examination of strategies to mitigate immunogenicity and off-target effects.

Main Results:

  • Chemically modified siRNAs demonstrate significant therapeutic potential.
  • Optimized siRNA design and modifications are crucial for efficacy.
  • Methods exist to manage and reduce adverse effects like immunogenicity and off-target activity.

Conclusions:

  • Chemically modified siRNAs represent a viable and advancing therapeutic strategy.
  • Careful design and modification are essential for successful RNAi-based drug development.
  • Addressing safety concerns is paramount for the clinical translation of siRNA therapeutics.