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    Gene Therapy

    Uploaded by

    Ritchel Ciprian

    Copyright:

    © All Rights Reserved
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    Gene Therapy

    Uploaded by

    Ritchel Ciprian
    32 views15 pages

    Copyright

    © © All Rights Reserved

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    PDF, TXT or read online from Scribd

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    Copyright:

    © All Rights Reserved
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    32 views15 pages

    Gene Therapy

    Uploaded by

    Ritchel Ciprian

    Copyright:

    © All Rights Reserved
    Download as PDF, TXT or read online from Scribd
    Download as pdf or txt
    of 15

    GENE THERAPY

    What is Gene Therapy ?

    = isinsertion of usually genetically


    altered genes into cells specially to
    replace defective genes in the
    treatment of genetic disorders to
    provide a specialized disease-
    fighting function.
    Category of Gene Therapy
    1. Somatic Gene Therapy- the process of
    DNA transfer involves any of the cells in the
    body except the sex cells.

    2. Germline gene therapy- results in


    permanent changes that are passed on to
    the patient’s children.
    Approaches to Gene Therapy
    1. GENE AUGMENTATION THERAPY-involves
    adding DNA that contains a functional version of
    the lost gene back into the cell.
    Ex. Cystic fibrosis
    Functioning Gene

    Insert
    gene

    • Cell with nonfunctioning cell functioning


    • Gene normally
    2. GENE INHIBITION THERAPY
    - use to transfer gene whose product
    either inhibits the expression of
    another gene or interferes with the
    activity of the product of another
    gene.
    -suitable for the treatment of
    infectious disease and cancer.
    3. KILLING OF SPECIFIC CELLS
    -insertion of a gene into a disease cell that
    causes the cell to die.
    It can be achieved by:
    1. Insertion of suicide gene that produces
    highly toxic product which ultimately kill the
    cell.
    2. Insertion of DNA that triggers the expression
    of a protein that marks the cell and
    subsequently attack by antibodies.
    Target Diseases of Gene Therapy
    • Severe Combined Immunodeficiency
    Disease(SCID)
    • Adenosine
    Deaminase(ADA)Deficiency-a genetic
    disorder that damages the immune
    system.
    • Blindness- inability to see
    • Hemophilia- Bleeder’s
    Disease( lack clotting factors)
    • Beta Thalassemia- hemoglobin
    synthesis disorder
    • Cancer
    • Cardio-vascular Disorders
    • September 14, 1990- the first
    successful trial of gene therapy
    was performed at the National
    Institute of Health Clinical Center.
    • The recipient is a 4-year old girl
    with ADA or Adenosine
    Deaminase Deficiency.
    • GENDICINE – the first commercial
    gene therapy that was approved by
    china for the treatment of certain
    cancer.
    • In December 7, 2011, Russian
    Ministry of Healthcare registered
    NEOVASCULGEN in the state registry
    of Medication. It is designed to treat
    a Peripheral Artery Disease.
    Risks of Gene Therapy
    • Chimeric Antigen receptor cell
    therapy (CAR-T)
    • For blood cancer patients
    • Side effect: swelling on the brain.
    Risks
    • Possibility of Harmful DNA mutation.
    • It may result to cancer.
    • Since viruses are used to deliver the
    functional copy of a gene, it may result to
    production of toxins, immune and
    inflammatory responses, gene control
    and targeting issues.
    Ethical and Moral Issues
    • Genetic Testing: Prenatal test- what if it
    will be found out that the embryo or
    fetus have mutated genes, that makes
    the individual abnormal, If you are the
    parent, will you continue to give birth to
    the child or abort so that he will not
    suffer in this life.
    Regulation on the use of gene Therapy

    • This may lead to the


    enhancement of other
    traits like height, beauty,
    intelligence, weight etc.
    Case Study:
    • Luxturna – the gene therapy for blindness
    specifically retinal diseases

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