Survey
* Your assessment is very important for improving the work of artificial intelligence, which forms the content of this project
Download Gene Therapy - Bluffton University
Transcript
Genetic Engineering of Bacteria to Produce Human Proteins Human insulin gene human cell bacterial DNA Bacteria produces human INSULIN protein, which is purified and given to patients Insert bacterial/human DNA into bacterial cell Genetic Engineering of Bacteria to Produce Human Proteins Human blood clotting gene human cell bacterial DNA Bacteria produces BLOOD CLOTTING PROTEIN, which is purified and given to patients Insert bacterial/human DNA into bacterial cell Genetic Engineering of Bacteria to Produce Human Proteins Human erythropoietin gene human cell bacterial DNA Bacteria produces ERYTHROPOIETIN PROTEIN, which is purified and given to patients Insert bacterial/human DNA into bacterial cell Gene Therapy • An experimental treatment which attempts to treat a disease by inserting a gene into the cells of a human • >3000 patients have been treated in >300 clinical trials around the globe • No gene therapy treatment is yet approved for general use Jesse Gelsinger - received gene therapy for OTC Deficiency - died September 1999 Gene Therapy for OTC Deficiency Normal OTC gene human cell Insert the OTC gene into a virus Deliver the virus directly into vein leading to liver virus infects liver cells and delivers gene OTC protein is produced inside the person Severe Combined Immunodeficiency (SCID) • The first disease to be treated with gene therapy • Persons with SCID lack a functional immune system and are susceptible to severe infections David Vetter (1972-1984) - lived in a sterile “plastic bubble” because had a single defective gene that caused Severe Combined Immunodeficiency (SCID) Gene Therapy for SCID Remove blood cells from patient Re-infuse the blood cells into the patient Infect blood cells with a virus that has been genetically engineered to carry the corrected SCID gene Ashanthi DeSilva - inherited SCID -received 1st ever gene therapy in 1990 Gene Therapy for SCID Using Long-lived Umbilical Cord Blood Cells Remove blood cells from the umbilical cord Re-infuse the cord blood cells into the patient Infect cord blood cells with a virus that has been genetically engineered to carry the corrected SCID gene Andrew Gobea (& mom) - inherited SCID - received 1st ever gene therapy using long-lived cord blood cells Other Partially Successful Gene Therapy Trials • therapy to induce new blood vessel growth in patients with blocked heart arteries or with other circulatory problems • therapy to induce brain tumor cells to “commit suicide” Ethical Questions Concerning Gene Therapy • Should treatments be used to change the appearance, intelligence, or athletic ability of healthy people? • Should parents be allowed to genetically manipulate their children before birth? • Should gene therapy be allowed that alters germ-line cells (reproductive cells that will pass the altered genes to future generations)?
