THE USE OF GENE THERAPY FOR IMMUNODEFICIENCY DISEASE

Abstract

Recent years have been characterized by important progress in biotechnology and molecular genetics. As a consequence, our knowledge of the genetic basis of human diseases and the possibilities of corrective gene transfer to mammalian cells have improved greatly. Gene transfer vectors based on viral systems have been investigated extensively and have been used to develop gene-based therapies for inherited and acquired human diseases. In particular, retrovirus-based gene transfer vectors have been used most frequently in current clinical gene therapy protocols because of their capability to stably integrate DNA material into the genome of the target cells. In 1990, through the use of retroviral-mediated gene transfer, two young girls affected by ADA-deficiency, were treated with infusions of gene-corrected autologous lymphocytes, making this form of inherited immunodeficiency the first disease to be treated by gene therapy. The results obtained from this and later clinical applications of gene therapy have indicated the possibilities of current gene transfer technologies and pointed the way to future applications for the treatment of other inborn errors of immunity, as well as a wider array of congenital and acquired human genetic diseases.