Gene Therapy

Gene therapy refers to a group of actual or future medical treatments that are aimed at changing the genetic structure of cells with defective genes that are causing disease. This may be attempted by trying to reverse the mutation of a gene, inserting a new genome into the genetic sequence, substitution of a normal gene for an abnormal (disease-causing) one and other techniques. The type of medical problem that can be remedied through this type of technology has customarily been untreatable until these advances were made. This includes genetic diseases, sickle cell thalassaemia, leukemia, and similar problems. Technical problems that have hampered the progress of gene therapy include the problems caused by the human immune system, which is designed to resist foreign intrusions into the body, the fact that some conditions result from multigenetic factors, thereby necessity multiple interventions, and the fact that therapies currently tend to be inherently short-term in nature and require substantial modifications to be made in order to become permanent.

In order to transfer the replacement gene into its desired position, it must be transported by what is known as a vector, which is customarily a virus that has been engineered for this purpose. The types of virus so far employed include adenovirus types similar to those causing the common cold, retrovirus and herpes simplex virus types. Of course, great care is necessary in dealing with this material. Various techniques have been developed for using the vector to transfer the genes, although practical difficulties mean that all remain complex and potentially expensive. Nevertheless, many people suffering from genetic disorders have had their hopes raised by the possibilities offered by gene therapy and some, for want of any alternative, are prepared to offer themselves as test subjects for unproven technologies. Theoretically, at least, the possibilities are immense.

Gene therapy is considered to present a number of ethical issues. First, it involves the issue of normality and the extent to which people with genetic disorders should be thought of as abnormal and to be cured. Second, the costs of the research are very high to the extent that the benefits of the research are likely to be rationed to the very rich. Third, the analysis and manipulation of cells, including stem cells, is considered by some to be unethical because of the ability of those cells to be part of an organism developing into an independent human being. Additional issues concern the extent to which human cells may be considered property on which experimentation may be conducted according to the dictates of the owners and the subsequent commercialization of products derived from such research. Attitudes toward these issues vary and in some cases have affected the location of firms undertaking research, which move away from states where their activities are unwelcome. Even so, commercialized products and techniques acceptable to national regulatory authorities have yet to appear.

Technological improvements have led to significant increases in the value and prospects of firms [Page 735]involved in providing commercial applications in the field. This has led to considerable investment in these firms and a number of attempts to sequester the possible financial advantages to be had from the technology through application of patents and other forms of intellectual property protection. This is controversial since many believe that any advances in medical technology should be made available to all the people of the world, albeit that some will add the provision of ability to pay. State governments are being required to formulate policy positions that connect both intellectual property and moral issues.

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