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Gene therapy: How does it work.

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Gene therapy is an experimental technique that uses genes to treat or prevent disease which allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

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Gene therapy: How does it work.

  1. 1. Gene Therapy by Prof. Liwayway Memije-Cruz
  2. 2. GENETHERAPY“It is tremendously rewarding to see how our work, starting with the test tube, can positively impact the society, by creating the groundwork for new medical treatments. “- Christine Karim
  3. 3. What Are Cells? • the basic building blocks of all living things. • the smallest structural and functional unit of an organism.
  4. 4. What are genes? • the basic physical and functional units of heredity. • made up of DNA. Some genes act as instructions to make molecules called proteins. • Genes vary in size from a few hundred DNA bases to more than 2 million bases. • provide the information for the production of specific proteins and enzymes that make muscles, bones, and blood, which in turn support most of our body’s functions, such as digestion, making energy, and growing.
  5. 5. What is Gene Therapy? • involves the manipulation of genes to fight or prevent diseases. • it introduces a "good" gene into a person who has a disease caused by a "bad" gene.
  6. 6. How does gene therapy work? What are the two types of gene therapy?
  7. 7. Somatic Gene Therapy • involves introducing a "good" gene into targeted cells to treat the patient • more conservative, safer approach because it affects only the targeted cells in the patient, and is not passed on to future generations. • used to correct the disease causing genetic sequence
  8. 8. Categories of Somatic Gene Therapy • ex vivo, which means exterior (where cells are modified outside the body and then transplanted back in again). In some gene therapy clinical trials, cells from the patient’s blood or bone marrow are removed and grown in the laboratory. The cells are exposed to the virus that is carrying the desired gene. The virus enters the cells and inserts the desired gene into the cells’ DNA. The cells grow in the laboratory and are then returned to the patient by injection into a vein. This type of gene therapy is called ex vivo because the cells are treated outside the body. • in vivo, which means interior (where genes are changed in cells still in the body). This form of gene therapy is called in vivo, because the gene is transferred to cells inside the patient’s body.
  9. 9. Ex vivo and In vivo
  10. 10. Germline Gene Therapy • which involves modifying the genes in egg or sperm cells, which will then pass any genetic changes to future generations.
  11. 11. Possible Effects of Gene Therapy • The viruses and other agents used to deliver the "good" genes can affect more than the cells for which they're intended. If a gene is added to DNA, it could be put in the wrong place, which could potentially cause cancer or other damage. • Genes also can be "overexpressed," meaning they can drive the production of so much of a protein that they can be harmful. • a virus introduced into one person could be transmitted to others or into the environment.
  12. 12. References: • https://kidshealth.org/en/parents/gene- therapy.html?fbclid=IwAR21RCVn4NR1CYtnOB u2Y9zih9bH2LfGZmdnk_YvzTC9EydrTuVuXn_ G_3w • https://www.fda.gov/consumers/consumer- updates/what-gene-therapy-how-does-it-work • https://jamanetwork.com/journals/jamaotolaryng ology/article-abstract/621891 • https://www.youtube.com/watch?v=GbJasFgJkL g&feature=emb_logo
  • EfrenGinAgustin

    Mar. 5, 2020
  • YanneTungol

    Feb. 29, 2020
  • RheaAngelaMores

    Feb. 20, 2020
  • memijecruz

    Feb. 19, 2020

Gene therapy is an experimental technique that uses genes to treat or prevent disease which allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

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