Related Experiment Video

Updated: Jun 6, 2026

Investigation of the Transcriptional Role of a RUNX1 Intronic Silencer by CRISPR/Cas9 Ribonucleoprotein in Acute Myeloid Leukemia Cells
09:16

Investigation of the Transcriptional Role of a RUNX1 Intronic Silencer by CRISPR/Cas9 Ribonucleoprotein in Acute Myeloid Leukemia Cells

Published on: September 1, 2019

[Downregulation of activated leukemic oncogenes AML1-ETO and RUNX1(K83N) expression with RNA-interference]

P V Spirin, F Baskaran, N N Orlova

    Molekuliarnaia Biologiia
    |November 25, 2010
    PubMed

    Insights

    Researchers utilized small hairpin RNAs (shRNA) to effectively reduce the expression of AML1-ETO and mutant RUNX1 (K83N) oncogenes in leukemic cells. This study demonstrates a novel gene silencing approach for potential therapeutic applications in leukemia.

    Area of Science:

    • Molecular Biology
    • Oncology
    • Gene Therapy

    Background:

    • AML1-ETO and mutant RUNX1 (K83N) are oncogenes frequently detected in leukemia cells.
    • Targeting these oncogenes is a potential strategy for leukemia treatment.
    • Gene silencing techniques offer a promising avenue for therapeutic intervention.

    Purpose of the Study:

    • To investigate the efficacy of small hairpin RNA (shRNA) in reducing the expression of AML1-ETO and RUNX1 (K83N) oncogenes.
    • To develop a lentiviral vector-based system for delivering shRNAs targeting these specific oncogenes.
    • To assess the stability and effectiveness of gene silencing in leukemic cells.

    Main Methods:

    • Design and synthesis of shRNAs targeting AML1-ETO and mutant RUNX1 (K83N).
    • Cloning of shRNAs into lentiviral vectors for efficient delivery.
    • Transduction of leukemic cells with lentiviral vectors expressing shRNAs.
    • Quantitative analysis of oncogene expression levels post-transduction.

    Main Results:

    • Successful design and expression of shRNAs targeting both AML1-ETO and RUNX1 (K83N).
    • Introduction of shRNAs via lentiviral vectors led to a significant and stable reduction in oncogene expression.
    • The siRNA approach demonstrated substantial gene silencing capabilities for these specific oncogenes.

    Conclusions:

    • shRNA delivered via lentiviral vectors is an effective method for stably reducing AML1-ETO and RUNX1 (K83N) expression in leukemic cells.
    • This gene silencing strategy holds potential for the development of novel therapeutic approaches for leukemia.
    • Further research is warranted to explore the in vivo efficacy and safety of this approach.

    More Related Videos

    Intracellular Phosphoflow Cytometry of Acute Myeloid Leukemia Patient-Derived Xenotransplants07:38

    Intracellular Phosphoflow Cytometry of Acute Myeloid Leukemia Patient-Derived Xenotransplants

    Published on: June 6, 2025

    Describing a Transcription Factor Dependent Regulation of the MicroRNA Transcriptome07:23

    Describing a Transcription Factor Dependent Regulation of the MicroRNA Transcriptome

    Published on: June 15, 2016

    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...
    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...
    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...
    Regulation of Expression at Multiple Steps01:23

    Regulation of Expression at Multiple Steps

    The gene expression in cells is regulated at different stages: (i) transcription, (ii) RNA processing, (iii) RNA localization, and (iv) translation. Transcriptional regulation is mediated by regulatory proteins such as transcription factors, activators, or repressors—these control gene expression by initiating or inhibiting the transcription of genes. Once a precursor or pre-mRNA is produced, it undergoes post-transcriptional modification, including 5' capping, splicing, and the addition of a...
    Abnormal Proliferation02:23

    Abnormal Proliferation

    Under normal conditions, most adult cells remain in a non-proliferative state unless stimulated by internal or external factors to replace lost cells. Abnormal cell proliferation is a condition in which the cell's growth exceeds and is uncoordinated with normal cells. In such situations, cell division persists in the same excessive manner even after cessation of the stimuli, leading to persistent tumors. The tumor arises from the damaged cells that replicate to pass the damage to the daughter...
    Regulation of Expression Occurs at Multiple Steps02:24

    Regulation of Expression Occurs at Multiple Steps

    Gene expression can be regulated at almost every step from gene to protein. Transcription is the step that is most commonly regulated. This involves the binding of proteins to short regulatory sequences on the DNA. This association can either promote or inhibit the transcription of a gene associated with the respective sequence.
    Transcription results in the generation of precursor (pre-mRNA) that consists of both exons and introns, which needs further processing before being translated to a...