Gene therapy for inherited disorders

With an increase in understanding of the role of genes in disease pathogenesis, gene therapy has become a reality for some disorders and an increased possibility for many others. One type of gene therapy requires only gene addition for treatment. Different methods for gene addition are being tested, based on the cell typology necessary to make the therapy effective. Gene therapy trials for patients with adenosine deaminase deficiency involve a retroviral approach. Gene therapy trials for patients with cystic fibrosis involve an adenoviral approach. This article describes the differences between these two methods of gene therapy. The application of this knowledge to pediatric nursing practice is discussed.