Understanding the complete genetic code might open the way to treating and curing various diseases that have remained untreatable for so many years. But with this good intent comes the possibility of using this technology with malevolent aims and unethical intent. The role of gene therapy as a key to prevention of disease through genetic engineering is often surrounded by this controversy.
The Role of Gene Therapy in Life-Threatening Diseases
The first trial involving gene therapy was conducted on a two-year-old girl suffering from severe immune disorder. The result was successful and had saved the life of the young girl. Furthermore, it had also saved the family from a treatment cost of $60,000 every year.
Another successful result of gene therapy was with sixteen cases of heart disease. Blood vessel growth was triggered by injecting copies of healthy genes directly to the heart. With the growth of new blood vessels around the clogged arteries, all the patients showed significant improvements.
With the cases above, it has to be noted that genetic engineering was only employed as a last option in fatal conditions.
One of the biggest setbacks to the field of gene therapy happened in September, 1999. A patient, Jesse Gelsinger, died as a test subject of gene therapy research at University of Pennsylvania. The reason for the death was severe immune response. From this incident, guidelines with the conduct of research and reporting procedures were re-evaluated with the intervention of congressional review. This slowed down the progress of research in the field of genetic engineering, particularly with its role in disease prevention.
In January, 2003, another setback gave a major blow to the application of gene therapy to prevent diseases. The FDA issued an order to stop all clinical trials involving gene therapy using retroviral vectors in blood stem cells. The reason for this move was that one test subject acquired a leukemia-like condition after undergoing a French gene therapy trial. The ban was only eased in April 2003.
The major challenge with the application of gene therapy is determining the best method to deliver the gene to the appropriate cells. Another challenge is finding the right method to incorporate the gene unto the target cell’s chromosome.
Another problem is the limitation of this technology. Correcting a genetic problem does not prevent it from happening with the descendants. The current procedures used in clinical research only targets the existing tissue with the individual to alleviate symptoms or prevent a disease. But this does not mean that it will prevent the disease with future offspring.
To accomplish the goal of disease prevention for the next generations, gene therapy would require targeting the germ cells, the sperm and the egg cells. The gene therapy methods only target the somatic cells or non-sexual cells for the purpose of preventing diseases. This method of targeting the herm cells poses different risks and technical problems. Furthermore, this has an ethical dilemma of making a genetic decision for the future generations without their consent.
The prospect of prevention of disease through genetic engineering may be beneficial to humankind but it poses several issues and limitations. As of the present, the use of gene therapy has not yet been successful except for a few cases. This technology is still on its infancy stage and not yet ready to be applied for the general public. In addition, researchers intend to employ this technology in curing and preventing fatal diseases in an attempt to avoid its abuse.
What will be the future of gene therapy for disease prevention? Only time will tell.