HUMAN GENETIC-DISEASE AND THE MEDICAL NEED FOR SOMATIC GENE-THERAPY

The introduction of the adenosine deaminase (ADA) gene into the mature lymphocytes of an ADA-deficient patient offers only a temporary treatment of the disease, but has paved the way for potential cure of this and other inherited diseases through the use of somatic gene therapy. With the increasing wealth of molecular information regarding many inherited and acquired disorders, somatic gene therapy as a therapeutic modality has become a reality. However, successful gene transfer depends not only on the molecular definition of a disease gene, but on the understanding of the underlying pathophysiology of the disease. An intimate knowledge of the biology of the target tissues for gene transfer is a requisite for the development of successful gene therapy. Progress in a variety of gene transfer strategies suggests that gene therapy may potentially offer a cure for inherited hematopoietic diseases, disorders of liver and muscle, as well as acquired diseases such as malignancies.