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Gene Therapy Report STS.pptx ...........
- 1. GENE THERAPY SCIENCE TECHNOLOGY AND SOCIETY ACP 1F
- 2. A. OVERVIEW Genes contain DNA - the code that controls much of the body's form and function. DNA controls everything from hair color and height to breathing, walking and digesting food. Genes that don't work properly can cause disease. Sometimes these genes are called mutations. Gene therapy - aims to fix a faulty gene or replace it with a healthy gene to try to cure disease or make the body better able to fight disease. It holds promise as a treatment for a wide range of diseases. Gene therapy is the use of genetic material to treat or prevent disease.
- 3. The U.S. Food and Drug Administration (FDA) has approved gene therapy products for several conditions, including cancer, spinal muscular atrophy, hemophilia and sickle cell disease. But for most people, gene therapy is available only as part of a clinical trial. Clinical trials are research studies that help healthcare professionals find out whether a gene therapy approach is safe for people. Clinical trials also help healthcare professionals learn how gene therapy affects the body.
- 4. B. TERMINOLOGIES Deoxyribonucleic acid (DNA)- is the material that exists in every cell in your body that holds your genetic code. It makes up your bodys instruction manual. Genes- are segments of DNA that determine our traits. Genes are the building blocks for your body. Every human has between 20,000 and 25,000 different genes, half of which are inherited from our biological mothers and the other half from our biological fathers. Chromosomes- are long, bundled strands of DNA, each of which contains many genes. In total, there are two sets of 23 chromosomes in a cell. Each set is inherited from our biological parents.
- 5. Gene therapy - seeks to alter genes to correct genetic defects and thus prevent or cure genetic diseases. Genetic engineering - aims to modify the genes to enhance the capabilities of the organism beyond what is normal.
- 6. C. TYPES OF GENE THERAPY There are two types of gene therapy: 1. In vivo (inside your body): Scientists put the vector carrying the new genes directly into your body via an injection or intravenous (IV) infusion 2. Ex vivo (outside your body): Scientists extract cells from your body and introduce them to the vector inside a petri dish. Then, your altered cells are returned to your body, where theyll hopefully multiply.
- 7. Each type has its own benefits: Benefits of in vivo therapies: Benefits of ex vivo therapies: Can deliver vectors across the body, which is useful for bone or blood diseases Better at targeting specific organs and cell types Quicker and less invasive Tend to pose fewer safety risks
- 8. D. DISEASES CURED BY GENE THERAPY Immunodeficiency disorders- involve malfunction of the immune system, resulting in infections that develop and recur more frequently, are more severe, and last longer than usual. Congenital blindness- is vision loss or severe vision impairment that is present at birth. Not included in this category are other types of blindness that develop because of injury or other medical conditions. Hemophilia- is a rare disorder in which the blood doesn't clot in the typical way because it doesn't have enough blood- clotting proteins (clotting factors).
- 9. Sickle cell anemia or sickle cell disease-affects the shape of red blood cells, which carry oxygen to all parts of the body. Red blood cells are usually round and flexible, so they move easily through blood vessels. In sickle cell anemia, some red blood cells are shaped like sickles or crescent moons. These sickle cells also become rigid and sticky, which can slow or block blood flow. Down syndrome- is a genetic disorder caused when abnormal cell division results in an extra full or partial copy of chromosome 21. This extra genetic material causes the developmental changes and physical features of Down syndrome.
- 10. E. BENEFITS OF GENE THERAPY: Fix genes that don't work properly- Faulty genes that cause disease could be turned off so that they no longer promote disease. Replace genes that don't work properly- Some cells become diseased because certain genes don't work properly or no longer work at all. Replacing these genes with healthy genes may help treat certain diseases. Make the immune system aware of diseased cells- In some cases, your immune system doesn't attack diseased cells because it doesn't see them as intruders. Healthcare professionals could use gene therapy to train your immune system to see these cells as a threat
- 11. F. RISKS OF GENE THERAPY: Unwanted immune system reaction- Your body's immune system may see the newly introduced viruses as intruders. As a result, it may attack them. This may cause a reaction that ranges from swelling to organ failure. Targeting the wrong cells- Viruses can affect more than one type of cell. So it's possible that the changed viruses may get into cells beyond those that aren't working properly. The risk of damage to healthy cells depends on which type of gene therapy is used and what it's used for. Infection caused by the virus- It's possible that once the viruses get into the body, they may once again be able to cause disease. Possibility of causing errors in your genes- These errors can lead to cancer.
- 12. Because gene therapy involves making changes to the bodys basic building blocks (DNA), it raises many unique ethical concerns. G.WHAT ARE THE ETHICAL ISSUES SURROUNDING GENE THERAPY? .
- 13. The ethical questions surrounding gene therapy and genome editing include: 1. How can good and bad uses of these technologies be distinguished? 2. Who decides which traits are normal and which constitute a disability or disorder? 3. Will the high costs of gene therapy make it available only to the wealthy?
- 14. 4.Could the widespread use of gene therapy make society less accepting of people who are different? 5.Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?
- 15. Current research on gene therapy treatment has focused on targeting body (somatic) cells such as bone marrow or blood cells. This type of genetic alteration cannot be passed to a persons children. Gene therapy could be targeted to egg and sperm cells (germ cells), however, which would allow the genetic changes to be passed to future generations. This approach is known as germline gene therapy.
- 16. The idea of these germline alterations is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known. Because people who would be affected by germline gene therapy are not yet born, they cant choose whether to have the treatment. Because of these ethical concerns, the U.S. Government does not allow federal funds to be used for research on germline gene therapy in people.
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