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Related Concept Videos

siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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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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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...
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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.
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Related Experiment Video

Updated: Dec 16, 2025

Bacterial Delivery of RNAi Effectors: Transkingdom RNAi
07:56

Bacterial Delivery of RNAi Effectors: Transkingdom RNAi

Published on: August 18, 2010

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Toxin-Mediated siRNA Delivery.

Spencer Zhao1, Jeffrey M Karp2, Nitin Joshi3

  • 1Center for Nanomedicine and Division of Engineering in Medicine, Department of Medicine, Brigham and Women's Hospital, Boston, MA 02115, USA.

Trends in Pharmacological Sciences
|July 5, 2020
PubMed
Summary
This summary is machine-generated.

Diphtheria toxin (DT) was engineered to deliver small interfering RNA (siRNA) into cells, demonstrating a novel method for nucleic acid delivery. This research expands the potential of AB toxins as intracellular delivery vehicles for gene-based therapeutics.

Keywords:
diphtheria toxinglioblastomasiRNA delivery

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Phage-mediated Delivery of Targeted sRNA Constructs to Knock Down Gene Expression in E. coli
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Area of Science:

  • Biochemistry
  • Molecular Biology
  • Drug Delivery Systems

Background:

  • AB toxins possess inherent cell-targeting and endosomal escape capabilities, making them promising for intracellular delivery.
  • The use of AB toxins for delivering nucleic acid-based therapeutics remains underexplored.
  • Diphtheria toxin (DT) is a well-characterized AB toxin with potential for therapeutic applications.

Purpose of the Study:

  • To investigate the feasibility of using diphtheria toxin (DT) for the functional delivery of small interfering RNA (siRNA).
  • To expand the application of AB toxins beyond traditional cytotoxic roles towards nucleic acid delivery.
  • To demonstrate the potential of engineered toxins as platforms for gene therapy.

Main Methods:

  • Conjugation of siRNA to diphtheria toxin (DT).
  • In vitro assessment of cellular uptake and functional delivery of siRNA by the DT-siRNA conjugate.
  • Evaluation of gene silencing efficacy mediated by the delivered siRNA.

Main Results:

  • Successful functional delivery of siRNA using diphtheria toxin (DT) in vitro.
  • Demonstration of siRNA's ability to induce gene silencing upon delivery by DT.
  • Confirmation of DT's compatibility with nucleic acid therapeutics for intracellular delivery.

Conclusions:

  • Diphtheria toxin (DT) can be effectively utilized as a vehicle for functional siRNA delivery.
  • This study represents a significant advancement in utilizing AB toxins for nucleic acid delivery.
  • Engineered diphtheria toxin offers a promising platform for developing novel gene delivery strategies.