Gene therapy shi.,


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Gene therapy shi.,

  1. 1. Alright tellme how you want tochange…!
  2. 2. • It is a technique for correcting defective genes that are responsible for disease development
  3. 3.  1. A normal gene inserted to compensate for a nonfunctional gene. 2. An abnormal gene traded for a normal gene 3. An abnormal gene repaired through selective reverse mutation 4. Change the regulation of gene pairs
  4. 4.  · 1970s Gene surgery proposal • 1980s Great momentum to GT • 1983 Gene therapy Lesch-Nyhan disease GT • In 1988 (OTA)US Differentiate in somatic & germ LINE gene therapy • September 14, 1990 ADA deficiency GT(W. French Anderson and colleagues)successful • IN 1991 US govt.$58 million for gene therapy research • Oct.1999.Jesse Jelsinger first fatality in human gene EXP :Multiple organ failure.(Ornithine Trancarbamylase Deficiency) •2002 X-SCID: Fatal Leukemia
  5. 5.  • The first gene therapy was performed on September 14th, 1990 • Ashanti DeSilva was treated for SCID • Sever combined immunodeficiency Doctors removed her white blood cells, inserted the missing gene into the WBC, and then put them back into her blood stream . • This strengthened her immune system • Only worked for a few months
  6. 6.  • A vector delivers the therapeutic gene into a patient’s target cell • The target cells become infected with the viral vector • The vector’s genetic material is inserted into the target cell • Functional proteins are created from the therapeutic gene causing the cell to return to a normal state
  7. 7.  Somatic gene therapy: a. Ex vivo gene therapy b. In vivo gene Germline therapy & embryonic gene therapy
  8. 8.  1. Germline gene therapy refers to the permanent transfer of a gene into sperm or egg cells. 2. Embryonic gene therapy refers to the permanent transfer of a gene into the cells of an early embryo, just after the sperm and egg unite .3. In both cases, the delivered gene would become a permanent part of cells in the resulting adult.
  9. 9.  • Calcium phosphate • Gold nanoparticles (1 to 3 micrometer) :800psi • Electroporation • Microinjection • Chitosan • Elevated temprature • DEAE dextran • Cationic liposome • Activated dendrimer
  10. 10. • Short Lived – Hard to rapidly integrate therapeutic DNA into genome and rapidly dividingnature of cells prevent gene therapy from long time– Would have to have multiple rounds of therapy• Immune Response– new things introduced leads to immune response– increased response when a repeat offender enters• Viral Vectors– patient could have toxic, immune, inflammatory response– also may cause disease once inside• Multigene Disorders– Heart disease, high blood pressure, Alzheimer’s, arthritis and diabetes arehard to treat because you need to introduce more than one gene• May induce a tumor if integrated in a tumor suppressorgene because insertional mutagenesis
  11. 11.  • Neurogenic disease may be cured e.g. Alzeimer & Parkinsons Disease (life threatening diseases) • Difference types of Cancer gene therapy trials may get success in future • Germ cell gene therapy trials may get success in curing hereditary diseases • Pharmacogenetics & Geneticular molecular biotechnology • Applicable in livestock species. • GT: cautiously & seriously evaluate