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  • Gene Therapy

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    Daphne Olipas
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    Gene therapy involves inserting a functional gene into a patient's cells to replace a defective gene or control gene expression. There are two main types of gene therapy - somatic gene therapy, which affects only certain cells and is not inherited, and germline gene therapy, which affects sperm and egg cells and can be passed to offspring. Gene therapy techniques include gene augmentation therapy to introduce a new gene where a protein is absent, gene inhibition therapy to suppress a defective gene, and targeting specific cells like cancer cells by inserting a suicide gene or gene to mark the cells. Challenges of gene therapy include targeting the right cells, avoiding immune response, ensuring the new gene does not disrupt other genes, high cost, and ethical issues.

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    Gene therapy involves inserting a functional gene into a patient's cells to replace a defective gene or control gene expression. There are two main types of gene therapy - somatic gene therapy, which affects only certain cells and is not inherited, and germline gene therapy, which affects sperm and egg cells and can be passed to offspring. Gene therapy techniques include gene augmentation therapy to introduce a new gene where a protein is absent, gene inhibition therapy to suppress a defective gene, and targeting specific cells like cancer cells by inserting a suicide gene or gene to mark the cells. Challenges of gene therapy include targeting the right cells, avoiding immune response, ensuring the new gene does not disrupt other genes, high cost, and ethical issues.

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    4 views2 pages

    Gene Therapy

    Uploaded by

    Daphne Olipas
    Gene therapy involves inserting a functional gene into a patient's cells to replace a defective gene or control gene expression. There are two main types of gene therapy - somatic gene therapy, which affects only certain cells and is not inherited, and germline gene therapy, which affects sperm and egg cells and can be passed to offspring. Gene therapy techniques include gene augmentation therapy to introduce a new gene where a protein is absent, gene inhibition therapy to suppress a defective gene, and targeting specific cells like cancer cells by inserting a suicide gene or gene to mark the cells. Challenges of gene therapy include targeting the right cells, avoiding immune response, ensuring the new gene does not disrupt other genes, high cost, and ethical issues.

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    © All Rights Reserved
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    GSTS | Saint Louis University (PH)

    Study guide, definitions & notes

    Gene Therapy
    Tuesday, 2 November 2021 5:01 am

    ➢ Video: gene therapy basics


    ○ Introduction/removal/change of DNA (part only)
    ○ Can prevent disease ganon
    ○ Delivery of gene through vectors
    ▪ Use of virus, pero walang infection para hndi magkasakit.
    ▪ In vivo, ex vivo approach
    ➢ How gene therapy works
    ○ Defective gene results
    ▪ Failure of protein production
    ▪ Production of a non-functional protein
    ▪ Over production of protein
    ○ Gene therapy
    ▪ Experimental treatment - insertion of gene to correct effects of a
    defective gene
    - To treat illness
    ○ Types of Gene therapy
    a. Somatic gt
    - Transfer of a gene to any cell of the body that does not produce
    sperms/eggs
    - Effects of therapy will not be passed to patient's children
    b. Germline gt
    - Transfer of gene to cells that reproduce
    - Effects passed to children

    ○ Gene therapy techniques


    a. Gene augmentation therapy
    - Used if protein is absent/non-functional
    • Cystic Fibrosis

    b. Gene inhibition therapy


    - Disease related cell = new gene is inserted to defective
    gene/activity of gene.
    • Cancer

    c. Killing of specific cells


    - Targets diseased cells
    • Cancer cells
    1. Insertion of suicide gene
    2. Insertion of gene that produces protein that marks the
    diseased cell

    GSTS Page 51
    diseased cell

    ➢ Challenges of gene therapy


    a. Targeting the right cells
    b. Avoiding the immune response
    c. Making sure that the delivered gene does not disrupt the other gene
    d. High cost of gt
    e. Moral and ethical issues

    GSTS Page 52

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