siRNA and Molecular Therapies
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Questions and Answers

How many base pairs are typically found in a siRNA molecule?

  • 10
  • 50
  • 30
  • 20 (correct)

    • What protein is responsible for binding to one strand of siRNA in the RNA-induced silencing complex (RISC)?

  • Ago2 (correct)
  • RISC1
  • RISC2
  • Ago1

    • What is the primary outcome of siRNA silencing a gene?

  • The prevention of protein production (correct)
  • The production of a mutated protein
  • The degradation of the DNA sequence for the target gene
  • The increase in protein production

    • What is the major advantage of siRNA technology for drug development?

    <p>It targets specific genes, allowing for highly targeted therapies (B)</p> Signup and view all the answers

    What is the role of the RISC complex in siRNA-mediated gene silencing?

    <p>Binding to and degrading target mRNA molecules (C)</p> Signup and view all the answers

    What are some of the target tissues for siRNA-based therapies?

    <p>Eyes, lungs, liver, skin, kidney and tumours (A)</p> Signup and view all the answers

    What is a significant challenge of siRNA technology in drug development?

    <p>The potential for off-target effects and immune responses (A)</p> Signup and view all the answers

    Who were the scientists awarded the Nobel Prize for their discovery of the process of siRNA?

    <p>Andrew Fire and Craig Mello (C)</p> Signup and view all the answers

    What are the two main drug targets discussed in the content?

    <p>Receptors and Enzymes (A)</p> Signup and view all the answers

    Which technology is NOT discussed in the content?

    <p>CRISPR (D)</p> Signup and view all the answers

    What is the primary goal of this presentation?

    <p>To introduce various molecule-based therapies and their market status (C)</p> Signup and view all the answers

    What is the purpose of the 'Market Story' sections in the presentation?

    <p>To provide an overview of the market potential and current state of each technology (A)</p> Signup and view all the answers

    Which of the presented technologies is described as a defense against viruses?

    <p>siRNA (C)</p> Signup and view all the answers

    What is the primary function of blocking peptides, as described in the content?

    <p>To disrupt the interaction between proteins by inhibiting their binding (A)</p> Signup and view all the answers

    What is the main purpose of discussing receptor trafficking and anchoring proteins?

    <p>To show how these proteins can be targeted by blocking peptides (B)</p> Signup and view all the answers

    How do blocking peptides differ from aptamers in their mechanism of action?

    <p>Blocking peptides are synthesized chemically while aptamers are selected by evolution (D)</p> Signup and view all the answers

    What is the key difference between siRNA's and aptamers' function?

    <p>siRNA targets specific genes while aptamers inhibit protein interactions (B)</p> Signup and view all the answers

    How do aptamers contribute to toxin removal?

    <p>By binding to and neutralizing toxins, preventing them from interacting with target cells (B)</p> Signup and view all the answers

    What is the essential role of transport proteins in controlling receptor expression?

    <p>They transport receptors to the cell surface for activation (A)</p> Signup and view all the answers

    Why is the postsynaptic density considered to be full of proteins?

    <p>Because it's the site of neurotransmitter release and signal transduction (B)</p> Signup and view all the answers

    How can blocking peptides be used as receptor activators?

    <p>By blocking the binding site of an inhibitory ligand (B)</p> Signup and view all the answers

    Why is the creation of blocking peptides considered a novel method to alter receptor function?

    <p>They offer a targeted approach to modulate receptor activity, unlike traditional drugs (D)</p> Signup and view all the answers

    What key benefit of blocking peptides is emphasized in the content?

    <p>They are highly specific and target only specific receptors (D)</p> Signup and view all the answers

    Which statement best describes the current market story for blocking peptides?

    <p>Blocking peptides are still under development and research, with only a few candidates in clinical trials (D)</p> Signup and view all the answers

    Which of the following drug types are NOT mentioned as major categories currently on the market?

    <p>Antibody inhibitors (D)</p> Signup and view all the answers

    What is the first biologic drug mentioned in the text?

    <p>Humanised insulin (D)</p> Signup and view all the answers

    Which protein is not a target of a biologic drug listed in the table?

    <p>EGF (B)</p> Signup and view all the answers

    According to the table, which drug is the most expensive?

    <p>Comirnaty (B)</p> Signup and view all the answers

    Which of the following is not a type of biologic drug mentioned in the text?

    <p>Small molecules (D)</p> Signup and view all the answers

    How does siRNA technology directly target the production of proteins?

    <p>By degrading mRNA molecules that code for the targeted protein (D)</p> Signup and view all the answers

    What is the primary function of DICER in the antiviral defense system?

    <p>It produces small interfering RNA (siRNA) from viral RNA (A)</p> Signup and view all the answers

    What is the ultimate outcome of the interaction between siRNA/RISC complex and mRNA?

    <p>The prevention of protein production (C)</p> Signup and view all the answers

    What is the main purpose of siRNA technology?

    <p>To inhibit the production of specific proteins (D)</p> Signup and view all the answers

    What is the role of mRNA in protein synthesis?

    <p>It carries the genetic code from DNA to the ribosomes where proteins are made (A)</p> Signup and view all the answers

    Which of the following describes the process of translation in protein synthesis?

    <p>The production of a protein based on the genetic code in mRNA (C)</p> Signup and view all the answers

    What is the role of post-translational modifications in protein synthesis?

    <p>They add or remove chemical groups to a protein, affecting its function (B)</p> Signup and view all the answers

    What is the primary goal of using siRNA technology in therapeutic applications?

    <p>To prevent the expression of disease-causing genes or proteins (C)</p> Signup and view all the answers

    How does the siRNA/RISC complex contribute to gene silencing?

    <p>It degrades the mRNA transcripts that code for the targeted protein (B)</p> Signup and view all the answers

    Which of the following is a potential advantage of using siRNA technology as a therapeutic strategy?

    <p>It can specifically target and silence disease-related genes (C)</p> Signup and view all the answers

    Flashcards

    siRNA

    Small interfering RNA used for gene silencing.

    Gene silencing

    The process of preventing gene expression or silencing a gene.

    RISC

    RNA-induced silencing complex that binds siRNA and mRNA.

    Ago2 protein

    A component of RISC that helps bind siRNA.

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    mRNA

    Messenger RNA that carries genetic information for protein synthesis.

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    Nobel Prize in 2006

    Awarded to Andrew Fire and Craig Mello for discovering siRNA.

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    Therapeutic areas of siRNA

    Tissue types and conditions targeted by siRNA therapy.

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    Commercial siRNA synthesis

    Service offered by companies to create siRNA sequences.

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    Molecular Drug Targets

    Components like DNA, RNA, and proteins targeted by drugs.

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    Biologics

    Biological products such as antibodies and proteins used as therapies.

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    First Biologic Drug

    Humanized insulin, introduced in 1982.

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    Approval of Biologics

    Increase in biologics approval, from 6 in 2010 to 15 in 2022.

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    Top Selling Drugs 2021

    In 2021, 12 of the top 20 drugs were biologics.

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    Transcription

    Process of converting DNA to mRNA.

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    Translation

    Process of synthesizing proteins from mRNA.

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    RISC Complex

    RNA-induced silencing complex that uses siRNA to silence mRNA.

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    Antiviral Defense

    Cellular response involving siRNA to combat viral RNA.

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    DICER

    Enzyme that cuts viral RNA into smaller fragments.

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    Post-translational Modification

    Changes made to proteins after translation that affect function.

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    Antibody

    A protein used by the immune system to identify and neutralize pathogens.

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    Receptor Agonists

    Drugs that activate receptors to produce a biological response.

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    Molecular-based Therapies

    Medical treatments that utilize molecular mechanisms, including siRNA, aptamers, and peptides.

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    Mechanism of Action

    The specific biochemical interaction through which a drug produces its effect.

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    Drug Targets

    Molecules, typically proteins, that drugs interact with to produce a therapeutic effect.

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    Receptors

    Protein molecules on cell surfaces that receive signals from drugs and hormones.

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    Aptamers

    Short, single-stranded RNA or DNA that binds specifically to target molecules.

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    Blocking Peptides

    Short proteins that inhibit or activate receptor function.

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    Transport Proteins

    Proteins that move substances across cell membranes.

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    Drug Labelling

    Using aptamers to tag drugs for targeting and tracking delivery.

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    Market Activity

    Analysis of the commercial landscape for specific drug technologies.

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    Benefits and Challenges

    Pros and cons associated with the use of a therapy or technology.

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    Toxin Removal

    Using aptamers to bind and remove harmful substances from the body.

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    Receptor Trafficking

    The process of moving receptors to and from the cell surface.

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    Study Notes

    Molecular-Based Therapies and Technologies: siRNA

    • siRNA (small interfering RNA) is a molecular-based technology used to inhibit protein production by targeting mRNA for destruction.
    • The process mirrors the body's antiviral defense. Viral RNA is cut into smaller pieces (siRNA) by DICER, which then bind to RISC (RNA-induced silencing complex). RISC binds to one strand of siRNA, and the complex targets corresponding mRNA. The targeted mRNA is cut, silencing the gene.
    • To use siRNA therapeutically, a specific siRNA sequence matching the target mRNA needs to be created. This is synthesized, then introduced to cells.
    • RISC binds to one strand and the siRNA/RISC complex binds the target mRNA, cutting it, and silencing the gene.
    • siRNA has been trialled in various tissues (eyes, lungs, liver, skin, kidney, tumors).Therapeutic areas explored include age-related macular degeneration (AMD), asthma, metabolic diseases, dermatology, renal failure, and cancers.

    Drug Targets and Biologics

    • Major drug targets are receptors and enzymes, but also DNA, mRNA, and proteins.
    • Traditional drugs include receptor agonists, antagonists, and enzyme inhibitors (small molecules).
    • Biologics (e.g., antibodies, proteins, peptides, DNA/RNA molecules, synthetic vaccines) are increasingly important. More biologics were approved in 2022 than in 2010.
    • Biologics, including antibodies and protein-based drugs, dominate the top 20 selling drugs (2021).
    • Humanized insulin was the first biologic drug (1982).

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    Description

    Explore the fascinating world of small interfering RNA (siRNA) and its role in molecular-based therapies. This quiz covers the mechanisms of action, therapeutic applications, and the processes involved in gene silencing. Delve into how siRNA is utilized in various medical conditions and its potential in future treatments.

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