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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...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
Modified-Release Drug Delivery Systems: Site-Targeted01:24

Modified-Release Drug Delivery Systems: Site-Targeted

Site-targeted drug delivery systems enhance therapeutic efficacy while minimizing systemic toxicity and treatment costs. Unlike conventional methods, these systems ensure precise drug delivery, improving bioavailability and reducing side effects. Targeted drug delivery is classified into three levels. First-order targeting directs drugs to the capillary beds of specific organs or tissues. Second-order targets specific cell types, such as tumor cells, using receptor-mediated interactions.
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...

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Related Experiment Video

Updated: Jun 2, 2026

Bacterial Delivery of RNAi Effectors: Transkingdom RNAi
07:56

Bacterial Delivery of RNAi Effectors: Transkingdom RNAi

Published on: August 18, 2010

Special delivery: targeted therapy with small RNAs.

D Peer1, J Lieberman

  • 1Laboratory of Nanomedicine, Department of Cell Research and Immunology, GS Wise Faculty of Life Science and Center for Nanoscience and Nanotechnology, Tel Aviv University, Tel Aviv, Israel. peer@post.tau.ac.il

Gene Therapy
|April 15, 2011
PubMed
Summary

Small RNA drugs offer new therapeutic targets by silencing disease genes. Overcoming delivery challenges is key to realizing their full potential for targeted gene silencing therapies.

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Last Updated: Jun 2, 2026

Bacterial Delivery of RNAi Effectors: Transkingdom RNAi
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06:53

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Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
12:03

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy

Published on: September 5, 2016

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Pharmacology

Background:

  • RNA interference (RNAi) enables gene expression knockdown.
  • Small RNA-based drugs hold promise for targeting disease-causing genes.
  • Effective delivery of small RNA therapeutics remains a significant challenge.

Purpose of the Study:

  • To review advancements in overcoming barriers to systemic, local, and cellular RNA drug delivery.
  • To highlight strategies for achieving targeted uptake of small RNA therapeutics.

Main Methods:

  • Review of recent scientific literature on RNA drug delivery systems.
  • Analysis of strategies for enhancing systemic, local, and cellular delivery.
  • Focus on methods promoting targeted cellular and tissue uptake.

Main Results:

  • Progress has been made in addressing systemic, local, and cellular delivery barriers.
  • Various strategies are emerging to improve targeted delivery and uptake of small RNAs.
  • Targeted uptake mechanisms are crucial for therapeutic efficacy.

Conclusions:

  • Advancements in delivery technologies are critical for the clinical translation of small RNA drugs.
  • Overcoming delivery hurdles will expand the therapeutic potential of RNA interference.
  • Targeted delivery strategies are essential for effective RNA-based gene silencing therapies.