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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.
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...
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...
Inhibitors of Viral Protein Synthesis01:30

Inhibitors of Viral Protein Synthesis

Protein synthesis is indispensable for viral replication, as viruses lack the cellular machinery required for this process and must hijack the host's translational apparatus. In response, host cells deploy a critical innate immune defense involving interferons, specialized cytokines that play a central role in inhibiting viral propagation.Upon viral detection, infected cells release interferons that bind to receptors on adjacent uninfected cells, activating the JAK-STAT signaling pathway and...
Inhibitors Of Virion Release01:25

Inhibitors Of Virion Release

Viral replication and dissemination rely on efficient mechanisms for host cell entry, genome replication, assembly, and release. Influenza viruses, such as types A and B, are negative-sense single-stranded RNA viruses with a segmented genome, that depend on two critical surface glycoproteins to carry out these processes: hemagglutinin (HA) and neuraminidase (NA). HA initiates infection by binding to sialic acid residues on the surface of host epithelial cells, facilitating receptor-mediated...

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Related Experiment Video

Updated: Jun 11, 2026

DNA Vector-based RNA Interference to Study Gene Function in Cancer
13:10

DNA Vector-based RNA Interference to Study Gene Function in Cancer

Published on: June 4, 2012

Viral vector-mediated RNA interference.

Linda B Couto1, Katherine A High

  • 1Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.

Current Opinion in Pharmacology
|July 13, 2010
PubMed
Summary

Viral vector delivery of RNA interference (RNAi) offers a promising therapeutic strategy for gene silencing. Research addresses challenges like off-target effects to enable safe and effective clinical applications.

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Gene Therapy

Background:

  • RNA interference (RNAi) is a natural gene silencing process with significant therapeutic potential.
  • Effective delivery of RNAi therapeutics to target tissues is crucial for clinical success.
  • Viral vectors offer efficient and sustained gene expression for RNAi applications.

Purpose of the Study:

  • To review the potential of viral vector-mediated RNAi for medical interventions.
  • To discuss the challenges and solutions associated with RNAi delivery.
  • To highlight the future prospects of RNAi in disease treatment.

Main Methods:

  • Review of existing literature on viral vector systems (AAV, lentivirus) for RNAi.
  • Analysis of studies demonstrating RNAi efficacy in disease models.

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Lentivirus Production
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Lentivirus Production

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Chitosan/Interfering RNA Nanoparticle Mediated Gene Silencing in Disease Vector Mosquito Larvae

Published on: March 25, 2015

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Last Updated: Jun 11, 2026

DNA Vector-based RNA Interference to Study Gene Function in Cancer
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DNA Vector-based RNA Interference to Study Gene Function in Cancer

Published on: June 4, 2012

Lentivirus Production
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Lentivirus Production

Published on: October 2, 2009

Chitosan/Interfering RNA Nanoparticle Mediated Gene Silencing in Disease Vector Mosquito Larvae
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Chitosan/Interfering RNA Nanoparticle Mediated Gene Silencing in Disease Vector Mosquito Larvae

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  • Examination of safety concerns and mitigation strategies for RNAi therapy.
  • Main Results:

    • Viral vectors like AAV and lentiviruses show promise for safe and efficient RNAi delivery.
    • Proof-of-principle for viral vector-mediated RNAi demonstrated in numerous animal models.
    • Identified challenges include off-target effects, immune responses, and miRNA pathway alterations.

    Conclusions:

    • Viral vector-mediated RNAi holds revolutionary potential for treating various diseases.
    • Ongoing research is actively addressing safety concerns to facilitate clinical translation.
    • Advancements in RNAi technology and vector design pave the way for new therapeutic approaches.