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    Asia Pacific College of Advanced Studies College Department Gec Sts

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    Ken lopez
    Gene therapy is an experimental technique that works to treat diseases by inserting functional genes into patients' cells to replace missing or mutated genes, and the first gene therapy treatment was performed in 1990 on a child with severe combined immunodeficiency. However, gene therapy still faces challenges such as producing long-lasting effects, avoiding immune responses, and safely delivering genes without risk of cancer.

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    Asia Pacific College of Advanced Studies College Department Gec Sts

    Uploaded by

    Ken lopez
    396 views4 pages
    Gene therapy is an experimental technique that works to treat diseases by inserting functional genes into patients' cells to replace missing or mutated genes, and the first gene therapy treatment was performed in 1990 on a child with severe combined immunodeficiency. However, gene therapy still faces challenges such as producing long-lasting effects, avoiding immune responses, and safely delivering genes without risk of cancer.

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    STS (BEED-BSED 3) - Lecture 12

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    Gene therapy is an experimental technique that works to treat diseases by inserting functional genes into patients' cells to replace missing or mutated genes, and the first gene therapy treatment was performed in 1990 on a child with severe combined immunodeficiency. However, gene therapy still faces challenges such as producing long-lasting effects, avoiding immune responses, and safely delivering genes without risk of cancer.

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    © All Rights Reserved
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    396 views4 pages

    Asia Pacific College of Advanced Studies College Department Gec Sts

    Uploaded by

    Ken lopez
    Gene therapy is an experimental technique that works to treat diseases by inserting functional genes into patients' cells to replace missing or mutated genes, and the first gene therapy treatment was performed in 1990 on a child with severe combined immunodeficiency. However, gene therapy still faces challenges such as producing long-lasting effects, avoiding immune responses, and safely delivering genes without risk of cancer.

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    ASIA PACIFIC COLLEGE OF ADVANCED STUDIES

    A.H. Banzon St., Ibayo, City of Balanga, Bataan


    COLLEGE DEPARTMENT

    GEC STS ​– BEED/BSED 3


    1​st​ Semester AY 2020-2021
    LECTURE 12

    Gene Therapy
    ❖ an experimental technique that uses ​genes​ to treat or prevent disease. In the future, this
    technique may allow doctors to treat a disorder by inserting a ​gene​ into a patient's cells
    instead of using drugs or surgery.
    ❖ It is a technique for correcting defective genes responsible for disease development.

    Gene
    ❖ Basic unit of heredity
    ❖ Carried on a chromosome
    ❖ Encode how to make a protein: DNA RNA proteins
    ❖ Proteins carry out most of life’s function
    ❖ Causes dysfunction of a protein when altered

    How it started?
    ❖ In the 1980s, Scientists began to look into gene therapy.
    ❖ They would insert human genes into a bacteria cell.
    ❖ Then the bacteria cell would transcribe and translate the information into a protein
    ❖ Then they would introduce the protein into human cells

    The First Case.


    ❖ The first gene therapy was performed on September 14th, 1990
    ❖ Ashanti De Silva was treated for SCID (Severe combined immunodeficiency)
    ❖ Doctors removed her white blood cells, inserted the missing gene into the WBC, and
    then put them back into her bloodstream.
    ❖ This strengthened her immune system

    Two Basic Types of Gene Therapy


    1. SOMATIC CELL GENE THERAPY
    ❖ Therapeutic genes transferred into the somatic cells.
    ❖ Eg. Introduction of genes into bone marrow cells, blood cells, skin cells etc.

    2. GERM LINE GENE THERAPY


    ❖ Therapeutic genes transferred into the germ cells.
    ❖ Eg. Genes introduced into eggs and sperms.

    Two approaches of Gene Therapy


    1. Ex vivo gene therapy:​- transfer of genes to cultured cells and reinsertion.
    How does it work?
    Step 1:​ Treatment begins with cells being removed from an infected person. These
    cells are then grown and multiplied in a laboratory.
    Step 2​: Therapeutic gene will be introduced into the cultured cells using a virus called
    Vector. The virus’s DNA is taken out and replaced with the one that treats the patient.
    Step 3:​ The genetically transformed cells are selected and reinjected into the patient’s
    body.

    2. In vivo gene therapy:​- direct delivery of genes into the cells of a particular tissue in
    the body.
    Success depends on:
    ❖ efficiency of the uptake of the therapeutic gene by the target cells
    ❖ intracellular degradation of the gene and its uptake by nucleus
    ❖ the expression capability of the gene

    Medicine​- one example of a scientific and technological innovation that made a breakthrough
    in the area of human health.
    Before aspirin was ever understood at the nano level, it had been known for its effects.
    Pain of various forms could be relieved and softened by this medicine. The aspirin was already
    an ‘old’ technology at the beginning of the 21st century. The more recent findings in the areas
    of nano and gene science can be incorporated into medicine.

    What We Should Know about Stem Cell Treatment in the Philippines


    (Article by Totel De Jesus)

    Manila, Philippines---In recent years, it’s common to hear beauty doctors in the Philippines
    offering stem cell therapy as if it is a newfound medical fountain of youth, the answer to all
    medical worries.

    In various media platform, there are bombardment of advertisements from various hospitals
    and clinics packaging stem cell therapy as “cure-all” remedy
    With such a medical breakthrough, it begs the question on how equipped we are in offering
    such services. We can't help but ask the parameters on distinguishing the real experts from
    seemingly fly-by-night clinics and medical centers.

    Filipino-American doctor-lawyer Samuel Bernal, an expert in the fields of regenerative


    medicine and regulatory law, said the public should take a lot of precautions.

    “Be careful. Number one, stem-cell therapy is not a ’cure-all’ (medical procedure).” he said
    in an interview with the INQUIRER a few weeks after receiving the 2012 Presidential Award
    for Filipino Individuals and Organizations Overseas at the Malacañang Palace.

    “The goal of stem-cell therapy is not a longevity-anti-aging program. Stem cell therapy is
    actually trying to allow the body to heal itself,” he said.

    Educated at Johns Hopkins’ and Harvard Medical School, Dr. Bernal is currently Professor
    Emeritus of Medicine at the University of California-Los Angeles, member of American
    Association of Clinical Oncologists, the American Association of Cancer Researchers, and a
    recognized cancer investigator with the US National Institutes of Health.
    As a lawyer, his legal expertise includes cases on medical malpractice, medical product
    liability, as well as Federal Drug Administration (FDA) and Federal trade Commission’s
    (FTC) regulations in the testing, approval, advertising, and marketing of health products.

    He explained stem cell treatment requires “analyzing at the molecular level what is causing
    the problem or (what could be done) to prevent the problem.”

    “Stem cells are just a small part of the molecular approach to medicine.”

    Suppose scientists would like to fashion a gene to counter cancer cell growth. After several
    steps of developing the technology, its success would still depend on its science and
    implementation. To achieve the goal, the harmful gene has to be found among the 46 such
    structures in the ordinary cells of the person. The specific part of the gene doing the problem
    should be identified. Afterwards, that section of the gene must be removed and replaced with
    the “correct” or developed gene part to complete the therapy. This process would then correct
    the misinformation encoded in the whole gene.

    The financial cost of this therapy is not trivial. Each of the steps mentioned requires expertise
    and technological skill. Therefore, gene therapy can be costly.

    Current Status

    ❖ FDA hasn’t approved any human gene therapy product for sale
    Reasons:

    ❏ In 1999, 18-year-old Jesse Gelsinger died from multiple organ failure 4 days after
    treatment for ornithine transcarbamylase deficiency.
    ★ Death was triggered by severe immune response to adenovirus carrier
    ❏ January 2003, halt to using retrovirus vectors in blood stem cells because children
    developed leukemia-like condition after successful treatment for X-linked severe
    combined immunodeficiency disease

    Problems with Gene Therapy

    ❖ Short Lived
    ❖ Immune Response
    ❖ Viral Vectors
    ❖ Multigene Disorders
    ❖ May induce a tumor if integrated in a tumor suppressor gene because insertional
    mutagenesis

    References:

    a. Science, Technology, and Society by Daniel Joseph McNamara, SJ, Vida Mia
    Valverde and Ramon Beleno III.

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