Gary's Gravy Gene Therapy By Kole Drumheller and Austin Dillard

Gene Therapy

What is Gene therapy? This is a technique that is designed to introduce new genetic material into cells to help fix abnormal genes or to make a new protein that will help the person. Gene inserted directly into a cell normally don't function so scientists have use vectors, which can be viral or non viral, that delivers the Gene in a way that it functions. Viral vectors uses viruses with therapeutic DNA to insert the DNA into the cell and then the cell starts using the therapeutic DNA. Non viral vectors include the injection of naked DNA through the Gene gun which shoots DNA coated gold particles directly into the cell using high pressured gas.

History of Gene Therapy The idea of Gene therapy was first conceptualized in 1972 by Friedmann and Roblin they cited Rogers as proposing the idea. The first approved use of Gene therapy was preformed in the United States in September 1990 by William French Anderson on a four year old with ADA-SCID. The effects were temporary but successful. The first person to die by Gene therapy was Jesse Gelsinger in 1999. He died four days after being injected because of the adenoviral vector had caused a massive immune response.

Current uses of Gene therapy In some countries like China, Russia, and Europe gene therapies are used commercially to treat a variety of diseases like cancer, peripheral artery diseases, and LPLD which causes pancreatitis. Some researchers are using Gene therapy on animals such as monkeys or mice to examine the effects and to see if it can be used in humans.

Pros and cons Pros it can be used to cure genetic problems like some cancers, diabetes and other genetic diseases it can be used to help people with infertility Cons Short lived nature in most methods cause this to be an ineffective method and requires multiple treatments. This causes immune responses which can be harmful to the person and this reduces the effectiveness of multiple treatments since the immune response can stop it more effectively. Viral vectors are hard to control Some diseases are caused by multiple variations in the same Gene and this can occur when injecting therapeutic DNA The cost is very a and one treatment can cost about 1.6 million.

Ethical concerns One type of Gene therapy is Germaine where the Gene is inserted at the gamete and zygote stages and this raises concerns with the child doesn't get a choice and what would happen to the child. If it goes wrong at this level then the child could be worse off then when it started by getting more diseases when born then just the one that they were trying to cure. The use of gene therapy for enhancement and not curing diseases, like gene doping in sports to make athletes better. At the moment we don't know what effects this could have on future generations and if this could lead to new unpredictable diseases.

Case study They have recently used Gene therapy to cure blood cancer. What they do is filter the patients blood to remove millions of white blood cells and altering them with a gene that targets the cancer, then they return the genes back to the patient. They have tested this on more then 120 with different blood types and bone marrow cancers and it worked on most of them curing them while others had worked for a little bit then relapsed.