Gene Ther 2003 May;10(10):835-43
Londei M, Quaratino S, Maiuri L.
Institute of Child Health, University College London, London, UK.
"Gene therapy (GT) is still at the experimental stage and some recent setbacks have cooled the potential use of this therapeutic tool even in life-threatening conditions. However, this therapeutic approach has a potential, which is not limited to disease for which we have not other option. There are increasing evidence that GT will be soon used in diseases that are not life threatening. One group of diseases that can benefit from GT is the autoimmune one. Several experimental animal models have indicated the efficacy (proof of principle) of GT. In the present review, we have addressed the possibility that even extremely benign autoimmune-like diseases such as Celiac Disease (celiac disease) might one day profit from this type of therapy. We further point that in conditions such as celiac disease, where the trigger is well known and the pathogenic cascade is relatively well defined, a situation not common in autoimmunity, we can even have a better situation where to explore and use GT to control disease initiation and progression. Once the risks that are still intrinsic to GT will have been reduced the therapeutic options we outline in the present review might not appear too far from reality."
"celiac disease is the prototype of diseases in which a clear role of antigen-specific T cells has been demonstrated and where their inhibition results in disease amelioration. The present therapeutic approach is the removal of the antigenic challenge: the gluten-free diet, which is effective if there is a strict compliance to the diet. It is, however, not always easy to follow such strict restrictions for all life and alternative approaches have to be considered. The use of GT is at the moment a remote hypothesis as celiac disease is a relatively benign condition, with a valid therapeutic approach and GT has intrinsic risks that have been highlighted recently. The scope of this review is, however, to indicate a future application of GT when, as they will, the present limitations and intrinsic risks of GT will be overcome."