Human Gene Therapy

Human gene therapy gene therapy is the use of genes to treat diseases. It represents a great leap in our approach to treating human diseases and will have a major impact on medicine over the next decade. William French Anderson, Michael Biase, and Ken Culver were first successful in gene therapy in humans in 1990. They developed a treatment for adenosine deaminase (ADA) deficiency, which is a serious combination of immunodeficiency, also known as "boy with foam disease".

The biology of human gene therapy is very complex and there are still many techniques that need to be developed before gene therapy is properly used and require a more fully understood disease. In addition, since gene therapy involves changing the genetic settings of the body, it raises many unique ethical problems. Scientific and ethical discussions on gene therapy began years ago, but the first approved human gene therapy clinical trial was not initiated until 1990. This clinical experience seems to have succeeded as it greatly improves the health and well-being of the few people treated during the trial. However, the success of treatment is temporary as patients continue to receive traditional treatment with gene therapy. This makes it difficult to determine the true effectiveness of gene therapy itself. It is different from the effect of a more traditional treatment.

Measuring the success of treatment is a challenge for gene therapy. Research is full of practical and ethical challenges. As with drug clinical trials, the purpose of clinical trials in human gene therapy is to determine whether treatment is safe and effective, how to treat it, and whether treatment is effective. Based on the severity of the disease (if it is a more severe disease, it is likely to be a suitable candidate for the experiment), the feasibility of the treatment, and the success rate of treatment based on the animal model, for the study Select disease. This seems very reasonable. But, if you or your child is seriously ill, there is no other treatment. How objective is your decision to participate in the study?