Gene therapy aimed for action on fetuses still in the womb is the major concern and research objective of a group of scientists in the United Kingdom. The ultimate goal is to modify the genes of the fetus prior to birth with this cutting-edge procedure.
Damaged or abnormal genes are replaced with new genes inserted in a non-threatening virus or a similar vector. Gene therapy has been successful in treating patients with hemophilia and the “bubble boy” disease. The novelty of the new procedure involves performing gene therapy before the patient is even born, as the rate of success in children and adults is low due to immune responses. Scientists believe that starting the therapy on fetuses would not allow for a strong immune response, as the immune system is not likely to be fully developed. Furthermore, the disease could be cured before it has had a chance to cause severe damage to the individual. For example, cystic fibrosis causes severe lung damage before birth. Implementing gene therapy before birth could likely stop the disease from manifesting and the damage would not be irreversible, as seen when doctors treat children and adults with the disease.
Scientists are unsure whether the genes injected in utero may affect the host mother. Fetal gene therapy is still in early stages of development. As such, scientists will be experimenting on fetal primates and mice for a few more years until entertaining the thought of experimenting on humans. Nevertheless, the implications for this therapy are enormous, as they could provide an entirely new disease-free generation in the years to come.
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When discussing the topic of gene therapy, there are many reservations and for many reasons. I took a class in human genetics last semester and we had an opportunity to discuss the pros and cons of gene therapy. I agree with you that it is very difficult to perform gene therapy in adult mostly because of our immune system and also because the chance that the vector inserts into the wrong place resulting cancer is pretty high especially since we don't know very much about targeting yet. People right now still prefer traditional treatments to gene therapy mostly because there hasn't been that many successful trials. There was a case several years ago where gene therapy resulted in the death of someone. Immediately after that their hasn't been that much progress. But now more researches into gene therapy are growing as we know more about the human genome. I know that gene therapy can also be used to treat some form of arthritis.
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