Human Gene Therapy

Human gene therapy deoxyribonucleic acid (DNA) was discovered by Avery et al in 1944. Avery considers DNA to be a major genetic material. Watson and Crick later discovered the DNA double helix structure. Leder et al. Decoded the trinucleotide code that identifies the amino acids that make up the protein. Molecular biology was born (Sokol, Gewirtz, 1996). In 1990, a 4 year old girl with severe combined immunodeficiency disease (SCID) was the first girl who received gene therapy.

The first approach to human gene therapy began in 1990. Gene therapy involves treating people suffering from disease by placing good genes appropriate for their system so that they can fight disease. This research focuses only on situations in which certain genes are missing in the human body or genes are not functioning properly. The gene is added by inserting DNA into the patient's cell. The success of gene therapy is limited by many factors. For example, a subject that is typically receiving treatment has multiple problems that may interact in some unknown way and that person's immune system may be for a new gene introduced There is. Since 1990, gene therapy has made little progress.

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?