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Related Concept Videos

Nonsense-mediated mRNA Decay02:27

Nonsense-mediated mRNA Decay

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The Upf proteins that carry out nonsense-mediated decay (NMD) are found in all eukaryotic organisms, including humans. Each protein has an individual role, but they need to work in collaboration. Upf1 is an ATP-dependent RNA helicase that unwinds the RNA helix. Because Upf1 can unwind any RNA, Upf2 and Upf3 are required to help Upf1 discriminate between nonsense and normal mRNAs.
Usually, Upf3 binds to an Exon Junction Complex (EJC) at mRNA splice sites. If a ribosome fully translates the mRNA,...
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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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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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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.
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Certain biochemical processes, such as embryonic development and cell growth regulation, depend on the repression of specific genes. DNA binding proteins known as eukaryotic transcription inhibitors regulate the repression of gene expression in eukaryotes. The presence of these inhibitors at the required location and time in the cell is triggered by the presence of hormones and additional signals from other cells.
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Related Experiment Video

Updated: Nov 7, 2025

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
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Antisense Oligos May Hit "Undruggable" YAP1

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    |April 30, 2021
    PubMed
    Summary

    A novel antisense oligonucleotide targets YAP1, a previously undruggable Hippo pathway protein. This therapeutic approach shows preclinical promise and is now in a phase I clinical trial.

    Area of Science:

    • Oncology
    • Molecular Biology
    • Drug Discovery

    Background:

    • The Hippo pathway regulates organ size and is crucial in cancer development.
    • YAP1 (Yes1-associated transcriptional coactivator) is a key effector protein in the Hippo pathway.
    • YAP1 has been historically challenging to target therapeutically due to its nature as a transcription coactivator.

    Purpose of the Study:

    • To evaluate the therapeutic potential of targeting YAP1 using an antisense oligonucleotide (ASO).
    • To assess the preclinical efficacy and safety of the YAP1-targeting ASO.
    • To advance this novel therapeutic strategy into early-stage clinical investigation.

    Main Methods:

    • Development of a specific antisense oligonucleotide designed to inhibit YAP1.
    • Preclinical studies in relevant cancer models to assess efficacy.

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  • Pharmacokinetic and pharmacodynamic assessments.
  • Initiation of a Phase I clinical trial for safety and tolerability evaluation.

    • Main Results:

    • The developed antisense oligonucleotide demonstrated significant preclinical promise in targeting YAP1.
    • YAP1 inhibition showed potential anti-cancer effects in preclinical models.
    • The agent is currently under evaluation in a Phase I clinical trial, indicating initial safety and tolerability in humans.

    Conclusions:

    • Antisense oligonucleotide technology offers a viable strategy to target previously "undruggable" proteins like YAP1.
    • Targeting YAP1 represents a promising new therapeutic avenue in oncology.
    • The ongoing Phase I trial will provide critical data on the clinical utility of this approach.