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Related Concept Videos

RNA Interference01:23

RNA Interference

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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.
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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Experimental RNAi02:15

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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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siRNA - Small Interfering RNAs02:30

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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.
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...
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Related Experiment Video

Updated: Mar 22, 2026

Investigation of the Transcriptional Role of a RUNX1 Intronic Silencer by CRISPR/Cas9 Ribonucleoprotein in Acute Myeloid Leukemia Cells
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Current attempts to implement siRNA-based RNAi in leukemia models.

Hasan Uludağ1, Breanne Landry2, Juliana Valencia-Serna3

  • 1Department of Chemical & Materials Engineering, Faculty of Engineering, University of Alberta, Edmonton, Canada; Department of Biomedical Engineering, Faculty of Medicine & Dentistry, University of Alberta, Edmonton, Canada; Faculty of Pharmacy & Pharmaceutical Sciences, University of Alberta, Edmonton, Canada.

Drug Discovery Today
|April 30, 2016
PubMed
Summary

RNAi therapy, using small interfering RNA (siRNA), offers a new way to target leukemia-causing genes. This review covers RNAi delivery methods and future strategies for more effective and safer leukemia treatments.

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Area of Science:

  • Hematology
  • Molecular Biology
  • Pharmacology

Background:

  • Leukemias originate from genetic mutations in hematopoietic stem/progenitor cells.
  • These mutations result in an overpopulation of abnormal, transformed blood cells.
  • RNA interference (RNAi) offers a mechanism to silence specific disease-driving genes.

Purpose of the Study:

  • To review RNAi-mediated therapy for leukemia.
  • To discuss novel delivery technologies for RNAi.
  • To explore future directions for enhanced efficacy and safety.

Main Methods:

  • Review of current literature on RNAi therapy in leukemia.
  • Analysis of in vitro and preclinical animal models.
  • Examination of molecular targets and delivery systems for RNAi.

Main Results:

  • RNAi, particularly with small interfering RNA (siRNA), can effectively downregulate specific oncogenic drivers.
  • Various delivery technologies are being developed to enhance RNAi efficacy.
  • Preclinical data support the potential of RNAi-based approaches.

Conclusions:

  • RNAi-based therapies show promise for treating leukemias.
  • Advancements in delivery systems are crucial for clinical translation.
  • Future research should focus on optimizing efficacy and safety profiles.