Gene therapy, or gene transfer, involves the introduction of normal genes into cells in order to repair them or modify their function. The techniques are being developed to treat a variety of medical disorders including diabetes. The research shows great promise but it is still at an early stage.
The Human Genome Project, which resulted in the mapping of the entire human genome, continues to provide the data necessary to interpret the genetic contribution about a number of medical disorders. Treatments using gene therapy have revolutionised the way we think about how we could treat or even cure diabetes. Genes coding for all kinds of messages can be introduced into cells by using modified viruses as carriers. These instructions to a cell can include a whole variety of modifications to its function, even to synthesise and secrete insulin.
With respect to making an immediate impact to clinical medicine, gene therapy offers us most optimism if the disease is caused only by one defective gene. In these examples, such as in cystic fibrosis or haemophilia, gene therapy to replace the defective gene could potentially eliminate the disorder. In Type 1 or Type 2 diabetes, where the disorder has resulted from multiple factors, gene therapy is still proving to be a very a useful tool in the untangling of a more complex situation. Genetic research aimed at finding a cure for diabetes is being actively explored by scientists in universities and research centres around the world
Gene therapy is not only being used to instruct cells to make insulin, it is also being used to address some of the problems associated with diabetic complications. Clinical trials have been assessing whether gene therapy using genes designed to stimulate repair cells in wounds could result in healing of diabetic foot ulcers. There are also clinical trials underway for gene therapy treatment of diabetic neuropathy.
It is not all a bed of roses though. A number of potential risks have already been identified with gene therapy. Major risks of using viruses as vectors to deliver gene therapy include the potential for the introduction of a new infection or the production of a dangerous immune system reaction in the host.
Gene therapy therefore, has to be considered to be in the early stage of development. A lot of additional research will still be required before the techniques can be translated into medical treatments for diabetes. Although the potential is enormous it is important to realise that whilst research breakthroughs in this area will attract great media interest it will be many years before safe medical treatments become feasible.