Concluding Comments

Despite the substantial progress in our understanding of the molecular events that underlie the pathology of the unstable repeat expansion diseases, we are only beginning to dissect the pathogenic complexity of these important conditions. It is clear that the study of animal models of these disorders is providing critical insights into these disorders, insights that will undoubtedly lead to therapies to prevent or to reverse the pathogenesis of these slowly developing disorders in the near future. We begin to explore the concepts relevant to the treatment of disease in the next chapter.