Gene Therapy Case Study

The first gene therapy procedure was done in 1990 by Dr William French Anderson on a four year old girl, Ashanthi who had the ADA-SCID disease. At first, she was treated with regular adenosine deaminase enzyme injections, which received positive results but soon her health began to worsen and another treatment was needed. The next procedure was the bone marrow transplant that could only be performed if a well suited donor was found, which did not happen for the young patient. If none of the two treatments were applicable, the only solution for the infected child was to be detached in an artificial and germ free habitat. This is why the ADA-SCID disease is often referred to as “the bubble boy disease” or “bubble babies” for the infected children.

After six months, her white blood cell count had matched the normality. However, the results of Ashanthi’s gene therapy was cut short. To ensure that her health remained in a good state, they continued her adenosine deaminase enzyme therapy while the gene therapy was going on. This costed the doctors difficulty with calculating the overall efficiency of the gene therapy and to do so, they had to pause Ashanthi’s ADA therapy. Her condition worsened which meant that gene therapy did not fully work on Ashanthi, and she resumed her therapy treatments. Trials were looked into from these results and Haematopoietic stem cells were investigated. They are a special type of stem cell that can be found in the body that can develop into all types of different blood cells. This meant that these stem cells could be turned into white blood cells with the adenosine deaminase gene and injected into people with Ashanthi’s condition. The results were favorable where white blood cells did make adenosine deaminase but only in small numbers. Scientists soon uncovered non-myeloablative conditioning, that involved Haematopoietic stem cells to be separated from the patient and engineered with the ADA

Gene therapy includes the modification to the body’s set of rules, which causes large quantities of people to raise many ethical discussions. Some of the concerns surrounding gene therapy involve the fret of the way good and bad utilisations of gene therapy are separated from one another, the decision-maker that determines which traits are of a normal state and which make up a disorder, if people should be permitted to use gene therapy to intensify their main human traits (as in tallness or shortness, athletic capability, intellect, etc.) without necessarily having a disorder to treat, the high prices of gene therapy that invites only the wealthy, and if international usage of gene therapy would make our society less forgiving of people who are dissimilar. Bone marrow or blood cells have been the main targets for treating people that need gene therapy. This kind of gene therapy can not be passed on to the offspring of the patient. However, gene therapy can be targeted at germ cells (eggs of the female and sperm cells of the male), which would permit the added gene to be transferred to the next generations, known as the approach of “germline gene therapy”. The whole aspect of germline gene therapy is a very sensitive issue. On one hand, it holds the potential to relieve future generations of a family from having a distinctive genetic disease, and on the other, it may cause the fetus