5.2.3 Gene Therapy

1. 5.2.3 Gene Therapy • explain the term gene therapy; • explain the differences between somatic cell gene therapy and germ line cell gene therapy; • discuss the ethical concerns raised by the genetic manipulation of animals (including humans), plants and microorganisms

2. Gene Therapy • Using genetic technology to treat genetic disorders • Getting a working copy of a gene into cells that contain dysfunctional copies of the gene

3. Somatic Cell Gene Therapy • Certain genes switched on and off in differentiated cells • Augmentation = adding functioning genes into the relevant specialised cells meaning that the protein can be made and the cell will function normally • Killing Specific Cells = making cancer cells express genes producing proteins that make their cells vulnerable to attack by the immune system for targeted cancer treatments

4. Germline Gene Therapy • Stem cells that could become any type of cell or a new human being • Germline therapy is changing the genes of the first few cells meaning that the organisms is different • Somatic cell therapy is changing genes on part of the body, however the disease can still be inherited as the gene still exists in every body cell and gametes • Germline gene therapy is altering the organisms genes before development into a fetus, however this is illegal in humans because they say: • New human disease could be created • Human evolution would be interfered with • Too many ethical and moral issues with changing a human genome

5. Issues Somatic Cell Gene Therapy • Getting genes into target cells is tricky • Treatment is short-lived and has to be repeated • Difficult to get the gene functioning in the genome • Only the actual patient affected Germline Gene Therapy • More straightforward to deliver gene into cell • All subsequent cells will have the functioning gene • Considered unethical • Genetic manipulation passed onto children- could be a good or bad thing

6. Xenotransplantation • 60% transplant patients die on the transplant list • We can now consider xenotransplantation= transplants from other species • Pigs have been genetically engineered so they do not produce an enzyme involved in organ rejection Problems • Different organ size • Pigs only live to 15, so organs may age quickly • Pigs body temperature is 39⁰C, our is 37⁰C • Animal welfare groups think killing animals for organs is wrong • Jews and Muslims cannot eat pork, so could not have transplants • Possible disease transfer between animals and humans

7. Genetic Manipulation • What is wrong with moving a gene from one organisms to another? • Selective Breeding is unnatural? • Media call food ‘Frankenfood’ but are not aware that we eat DNA all the time and think that it will become part of out DNA if we eat it... • There were objections to the first vaccinations, the first organ transplant, IVF babies...