The Pros And Cons Of Human Gene Therapy

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Beginning with the first officially sanctioned human-gene therapy trials, there have been disappointing setbacks and great breakthroughs (Donegan).The success and failure rate of germline gene therapy seems to fluctuate because it is ever changing and improving. Most often times than not it does have a higher failure rate due to unwanted side effects that are life-threatening, or it does not “cure” the defective genome. The success and failure rate is ever-changing for the fact that some countries do not allow certain germline gene therapy testing, such as for the embryo. With that in mind it is hard to pinpoint an exact number of cases that passed or failed due to restricted testing protocols in different countries. Future success and failure …show more content…

The vectors are viruses that have had the infectious gene stripped out, the virus vectors include retroviruses, Adeno-associated viruses, and Herpes simplex viruses. The virus is mixed with cells extracted from the patient and then injected back into that patient. Viruses wrapped in a special protein coat are used because they are like genes, and will bind to the surface of cells. When the viruses are injected back into the body, they force their way into the cells (Human Gene. There are two main methods, the first method is to treat adult sperm and egg cells so the genetic defect is not passed on to their offspring. The second germline method is to treat what is known as a pre-embryo that may harbor an unwanted genetic default before it is implanted into the uterus through in-vitro (Human Gene). Germline Gene Therapy can be done by the insertion of a healthy gene into the fertilized egg, the changes in the gene will descend to every other cell in the offspring. However, three technical problems are preventing consideration of this technique for the use in human beings. "The first is that scientists have no way of diagnosing genetic disorders in the fertilized egg. Secondly, the procedure is most often used to insert genes into fertilized eggs - injection with a microscopically guided glass needle - has a high failure rate and thirdly, the problem is lack of control over where the gene is inserted into the embryo's genetic machinery” (Human). The sole purpose of doing germline gene therapy to an embryonic organism is to prevent any health issues before birth. An option for those who are at risk of procreating a child with cystic fibrosis, germline therapy could be done to alter the embryos DNA genome to create a healthy baby. Before an embryo is even developed some doctors and