ABSTRACT
INTRODUCTION The advent of new techniques in gene therapy gives hope to many patients suffering from diseases that are difficult to cure. Until now, the scope of gene therapy has been limited to a refractory disease because of safety issues, effective-ness, and long-term adverse effects. Although a small number of patients benefit from gene therapy, the recent revelation of pathogenesis, the development of new technology and tools, and a huge research activity will enable us to treat genetic and acquired diseases with gene therapy.
