RNA-Based Therapeutics and Genome Editing Quiz
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Questions and Answers

What is Macugen?

  • An aptamer that binds vascular endothelial growth factor (correct)
  • A genome-editing tool that targets and cleaves specific DNA sequences
  • A bacterial immune system that confers immunity in three steps
  • An RNA sequence that functions as a catalyst to cleave specific mRNA structures

    • What is the function of ribozymes?

  • To confer immunity in three steps
  • To edit specific DNA sequences
  • To cleave specific mRNA structures (correct)
  • To bind a specific target molecule with high specificity and binding affinity

    • What is the function of CRISPR/Cas9?

  • To bind a specific target molecule with high specificity and binding affinity
  • To cleave specific mRNA structures
  • To confer immunity in three steps
  • To edit specific DNA sequences (correct)

    • What are the two monogenic diseases that CTX001 is currently undergoing late-stage clinical trials for?

    <p>Transfusion-dependent β-thalassemia and Sickle Cell Disease</p> Signup and view all the answers

    What is the function of CTX001?

    <p>To elevate patient's HbF</p> Signup and view all the answers

    What are aptamers?

    <p>Single-stranded DNA or RNA-based sequences that fold up to adopt a unique 3D structure</p> Signup and view all the answers

    What is the potential use of aptamers?

    <p>For affinity-based purification, diagnostics, and therapeutics</p> Signup and view all the answers

    What is the function of ribozymes?

    <p>To cleave specific mRNA structures</p> Signup and view all the answers

    What is Macugen?

    <p>An aptamer that binds vascular endothelial growth factor</p> Signup and view all the answers

    What is the function of CRISPR/Cas9?

    <p>To edit specific DNA sequences</p> Signup and view all the answers

    What are the two monogenic diseases that CTX001 is currently undergoing late-stage clinical trials for?

    <p>Transfusion-dependent β-thalassemia and Sickle Cell Disease</p> Signup and view all the answers

    What is the function of CTX001?

    <p>To elevate patient's HbF</p> Signup and view all the answers

    What are aptamers?

    <p>Single-stranded DNA or RNA-based sequences that fold up to adopt a unique 3D structure</p> Signup and view all the answers

    What is the potential use of aptamers?

    <p>For affinity-based purification, diagnostics, and therapeutics</p> Signup and view all the answers

    What are the three classes of stem cells?

    <p>Totipotent, pluripotent, and multipotent</p> Signup and view all the answers

    What is the source of induced pluripotent stem (iPS) cells?

    <p>Non-pluripotent cells</p> Signup and view all the answers

    What is the advantage of adult stem cells over embryonic stem cells?

    <p>Adult stem cells can differentiate to yield major cell types</p> Signup and view all the answers

    What is somatic cell nuclear transfer (SCNT)?

    <p>A process to create new cell lines from non-pluripotent cells</p> Signup and view all the answers

    What is the main limitation of embryonic stem (ES) cells?

    <p>They have difficulties maintaining lines</p> Signup and view all the answers

    What is the process of generating induced pluripotent stem (iPS) cells?

    <p>Introducing ES-specific genes into non-pluripotent cells</p> Signup and view all the answers

    What is the difference between totipotent and pluripotent stem cells?

    <p>Totipotent stem cells can differentiate into any cell type, while pluripotent stem cells can differentiate into only some cell types</p> Signup and view all the answers

    What are the three classes of stem cells?

    <p>Totipotent, pluripotent, and multipotent</p> Signup and view all the answers

    What is the source of induced pluripotent stem (iPS) cells?

    <p>Non-pluripotent cells</p> Signup and view all the answers

    What is the advantage of adult stem cells over embryonic stem cells?

    <p>Adult stem cells can differentiate to yield major cell types</p> Signup and view all the answers

    What is somatic cell nuclear transfer (SCNT)?

    <p>A process to create new cell lines from non-pluripotent cells</p> Signup and view all the answers

    What is the main limitation of embryonic stem (ES) cells?

    <p>They have difficulties maintaining lines</p> Signup and view all the answers

    What is the process of generating induced pluripotent stem (iPS) cells?

    <p>Introducing ES-specific genes into non-pluripotent cells</p> Signup and view all the answers

    What is the difference between totipotent and pluripotent stem cells?

    <p>Totipotent stem cells can differentiate into any cell type, while pluripotent stem cells can differentiate into only some cell types</p> Signup and view all the answers

    What are pharmabiotics?

    <p>Drugs that combine pharmaceutical and probiotic components</p> Signup and view all the answers

    What are the potential applications of pharmabiotics?

    <p>Gastroenterology, immunology, and neurology</p> Signup and view all the answers

    What are the benefits of pharmabiotics?

    <p>Improving the efficacy of drug therapies, reducing side effects, and enhancing the gut microbiome's diversity and health</p> Signup and view all the answers

    What are the potential risks of pharmabiotics?

    <p>Allergic reactions, overgrowth of certain bacterial species, and antibiotic resistance</p> Signup and view all the answers

    What is the regulatory framework for pharmabiotics?

    <p>It varies depending on the country and region</p> Signup and view all the answers

    What are pharmabiotics?

    <p>Drugs that combine pharmaceutical and probiotic components</p> Signup and view all the answers

    What is the potential application of pharmabiotics?

    <p>Gastroenterology, immunology, and neurology</p> Signup and view all the answers

    What are the potential benefits of pharmabiotics?

    <p>Improving the efficacy of drug therapies, reducing side effects, and enhancing the gut microbiome's diversity and health</p> Signup and view all the answers

    What are the potential risks of pharmabiotics?

    <p>Allergic reactions, overgrowth of certain bacterial species, and antibiotic resistance</p> Signup and view all the answers

    What are the mechanisms of action of pharmabiotics?

    <p>Not yet fully understood, but thought to interact with the gut microbiome, immune system, and host cells</p> Signup and view all the answers

    Study Notes

    Overview of RNA-based Therapeutics and Genome Editing

    • Ribozymes are RNA sequences that function as catalysts to cleave specific mRNA structures, and can be directed to a specific mRNA by introducing short flanking oligos.
    • Aptamers are single-stranded DNA or RNA-based sequences that fold up to adopt a unique 3D structure, allowing them to bind a specific target molecule with high specificity and binding affinity.
    • Macugen is an FDA-approved aptamer that binds vascular endothelial growth factor and is indicated for the treatment of neovascular age-related macular degradation.
    • CRISPR-Cas9 is a bacterial immune system that confers immunity in three steps: acquisition, expression, and interference.
    • CRISPR-Cas9 can be exploited as a genome-editing tool by using a single-guide RNA molecule to target and cleave specific DNA sequences.
    • Editing events occur when the cell tries to repair the damage via either non-homologous end-joining or homology-directed repair.
    • CTX001 is a promising CRISPR/Cas9-based genome-editing therapy currently undergoing late-stage clinical trials for two monogenic diseases: Transfusion-dependent β-thalassemia and Sickle Cell Disease.
    • Both diseases result from mutations to the β-globin gene, and CTX001 functions to elevate patient’s HbF, thereby alleviating transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients.
    • CTX001 is co-run by Vertex Pharmaceuticals and CRISPR Therapeutics, founded in 2013 by Emmanuelle Charpentier.
    • Ribozymes have been targeted against hepatitis C, SARS-CoV-2, and Influenza in preclinical trials.
    • Aptamers may prove useful for affinity-based purification, diagnostics, and therapeutics, similar to antibodies.
    • CRISPR/Cas9-based genome-editing therapies have potential for treating a wide range of genetic diseases.

    Types of Stem Cells and Their Characteristics

    • There are three classes of stem cells: totipotent, pluripotent, and multipotent.
    • Stem cells are classified based on their source: embryonic, adult, and induced pluripotent (iPS) cells.
    • Embryonic stem (ES) cells are derived from pre-implant-stage human embryos, usually from in vitro fertilization procedures.
    • ES cells can multiply while remaining undifferentiated, but have limitations such as difficulties maintaining lines and potential for tumor formation.
    • Somatic cell nuclear transfer (SCNT), also known as therapeutic cloning, is a process to create new cell lines, but it has ethical and technical issues.
    • Adult stem cells are undifferentiated cells found in tissues or organs, can differentiate to yield major cell types, and are used to maintain and repair tissue.
    • Adult stem cells have advantages over ES cells, such as overcoming ethical difficulties and allowing for autologous transplantation.
    • Adult stem cells have been used to successfully treat over 60 human diseases.
    • Induced pluripotent stem (iPS) cells are artificially derived from non-pluripotent cells by inducing the expression of certain genes.
    • iPS cells are typically derived through transfection of stem cell-associated genes into non-pluripotent cells using viral vectors.
    • iPS cells have similarities to ES cells, but their generation has technical challenges such as inefficient process and genomic instability.
    • The process of generating iPS cells involves isolating and culturing host cells, introducing ES-specific genes into the cells, and harvesting and culturing the cells to generate iPS cells.

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    Description

    Test your knowledge on RNA-based therapeutics and genome editing with this informative quiz. From ribozymes to aptamers to CRISPR-Cas9, this quiz covers the basics of these technologies and their potential applications in treating genetic diseases. Learn about the FDA-approved aptamer Macugen, the promising CRISPR/Cas9-based therapy CTX001, and the preclinical trials targeting hepatitis C, SARS-CoV-2, and Influenza with ribozymes. Take

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