Scribd Logo
Upload

Welcome to Scribd!

  • 2K views

    Gene Therapy

    Uploaded by

    konuanlatimi
    By the end of the course, students will be able to define gene therapy, understand gene transfer terminology, and realize the significance of gene therapy research. Gene therapy introduces new genetic material into cells for therapeutic purposes by correcting defective genes responsible for diseases. It can replace abnormal genes with normal ones or repair genes to return them to normal function.

    Copyright:

    © All Rights Reserved
    Download as PPTX, PDF, TXT or read online from Scribd

    Gene Therapy

    Uploaded by

    konuanlatimi
    2K views85 pages
    By the end of the course, students will be able to define gene therapy, understand gene transfer terminology, and realize the significance of gene therapy research. Gene therapy introduces new genetic material into cells for therapeutic purposes by correcting defective genes responsible for diseases. It can replace abnormal genes with normal ones or repair genes to return them to normal function.

    Original Description:

    gen terapisi gene therapy

    Original Title

    gene therapy

    Copyright

    © © All Rights Reserved

    Available Formats

    PPTX, PDF, TXT or read online from Scribd

    Did you find this document useful?

    Is this content inappropriate?

    By the end of the course, students will be able to define gene therapy, understand gene transfer terminology, and realize the significance of gene therapy research. Gene therapy introduces new genetic material into cells for therapeutic purposes by correcting defective genes responsible for diseases. It can replace abnormal genes with normal ones or repair genes to return them to normal function.

    Copyright:

    © All Rights Reserved
    Download as PPTX, PDF, TXT or read online from Scribd
    Download as pptx, pdf, or txt
    2K views85 pages

    Gene Therapy

    Uploaded by

    konuanlatimi
    By the end of the course, students will be able to define gene therapy, understand gene transfer terminology, and realize the significance of gene therapy research. Gene therapy introduces new genetic material into cells for therapeutic purposes by correcting defective genes responsible for diseases. It can replace abnormal genes with normal ones or repair genes to return them to normal function.

    Copyright:

    © All Rights Reserved
    Download as PPTX, PDF, TXT or read online from Scribd
    Download as pptx, pdf, or txt
    of 85
    At a glance
    Powered by AI
    The key takeaways are that gene therapy involves introducing new genetic material into cells for therapeutic purposes by correcting defective genes responsible for diseases. There are different approaches for correcting faulty genes such as inserting a normal gene or swapping an abnormal gene.

    Some of the hallmarks of successful gene delivery are targeting the right cells, activating the gene so the produced protein functions properly, integrating the gene into the host cell's genetic material, and avoiding harmful side effects.

    The two main types of gene therapy are germ-line gene therapy which involves introduction of genes into germ cells, and somatic gene therapy which involves introduction of genes into somatic cells.

    Objectives of this lecture

    By the end of this course you will


    be able to:
    1. Define the term Gene Therapy
    2. Use the correct terminology for gene
    transfer
    3. Realize the significance of gene
    therapy research

    What is gene therapy?

    What is gene therapy?


    Introduction of new genetic material
    into a cell for therapeutic purposes
    Genetic Materials:
    DNA
    RNA

    What is Gene Therapy ?


    Gene therapy is a technique for correcting
    defective genes responsible for disease
    development.
    Researchers may use one of several approaches
    for correcting faulty genes:

    A normal gene may be inserted into a nonspecific location


    within the genome to replace a nonfunctional gene. This
    approach is most common.
    An abnormal gene could be swapped for a normal gene
    through homologous recombination.

    What is Gene Therapy ?


    o

    The abnormal gene could be


    repaired through selective reverse
    mutation, which returns the gene to
    its normal function.
    The regulation (the degree to which
    a gene is turned on or of) of a
    particular gene could be altered

    DNA

    RNA

    ATCG

    AUCG

    More stable in nucleases

    Less stable in nucleases

    Less stable in UV light

    More stable in UV light

    What are some of the hallmarks of


    successful gene delivery?
    TARGETING the right cells.
    How can one ensure that the gene gets into the correct cells?
    ACTIVATING the gene.
    For gene delivery to be successful, the protein that is
    produced must function properly.
    INTEGRATING the gene in the cells.
    Gene should integrate into, or become part of the host cell's
    genetic material, or that the gene finds another way to survive
    in the nucleus without being trashed.
    AVOIDING harmful side efects.
    If the body develops immunity against a specific gene delivery
    vehicle, future rounds of the therapy will be ineffective

    Disease-Causing Proteins

    Problems:
    Defect in protein expression
    Expression of harmful proteins

    Example:
    Glucose-6-phosphate dehydrogenase deficiency
    Cancer

    Solutions:
    Introduce a correct version of the gene
    Inhibit or block the harmful proteins

    Advantages of gene
    therapy

    Specificity
    Can be either temporary or
    permanent
    Localization
    Low immunogenicity

    Gene Therapy

    Germ-Line Gene Therapy:


    Introduction of genes into germ cells
    (sperm and ovum)

    Somatic Gene Therapy:


    Introduction of genes into somatic cells

    Germ-Line Gene Therapy

    Hypothetically, germ-line therapy


    prevents transfer of defective genes to
    subsequent generation

    Due to ethical and safety reasons, it is not


    presently accepted for human application

    However, it is used for the transgenic


    laboratory animals production

    Advantage

    Disadvantage

    Specificity

    Time and labor consuming

    High transfection efficiency

    Invasive
    Contamination
    Not every cell type is growable

    Ex vivo gene therapy

    Most widely used in clinical trials


    Some attempts in practice

    Advantage

    Disadvantage

    Low cost

    None
    specificity

    None invasive

    Low
    transfection
    efficiency

    Less

    In vivo gene therapy

    Some organs are less suited for ex


    vivo e.g. (brain, heart and lungs)
    More practical approach

    Gene Therapy
    Considerations

    What gene will be delivered (or targeted)?


    What method will be used?

    Elements for gene


    transfer

    Genetic material either for gene expression


    or downregulation
    Vector to carry the genetic material inside
    the cells

    Expression Vector
    (Plasmid)

    Viral Vector

    Non-Viral Vector

    Gene Transfer

    Transformation: introduction of genetic materials


    into bacteria

    Transfection: introduction of genetic materials into


    eukaryotic cells (e.g. fungi, plant, or animal cells)

    Transduction: introduction of genetic materials


    using viruses

    Lipofection: introduction of genetic materials using


    liposomes

    2
    8
    4

    You might also like