HPRT Gene Transfer as a Model for Gene Therapy

Abstract

One of the most striking features of modern molecular biology and of much of current medical research is the degree to which many of their common interests and techniques have converged. More than ever before, the most advanced and current methods of the biological sciences are being used in directly applied medical studies, and the clinical problems of medicine are becoming research interests for the most rigorous and highly trained basic scientists. The result is the development of conceptually new and effective approaches to an understanding of the pathogenesis, diagnosis and screening, prevention and therapy of the many human diseases that carry a genetic component (1). During the past several decades, techniques have become available to identify biochemical and metabolic defects responsible for many important disorders, to isolate and characterize the causative mutant genes and to identify genetic defects precisely at the level of the nucleotide sequence.