The use of gene therapy for immunodeficiency disease

Gene therapy has been thought of as the ultimate form of treatment for inherited diseases. The concept of correcting the fundamental genetic errors leading to human diseases is certainly more appealing than attempts to treat the infectious, metabolic, and other undesirable consequences of defective gene function. In the last decade, gene therapy has moved from that speculation to early clinical applications with clinical gene transfer protocols applied to a variety of disorders including inherited genetic defects, malignancies, and infectious diseases. Inborn errors of immunity have played a key role in the development of these early gene therapy strategies and have paved the way to a broader application of this novel branch of medicine. In this article, we summarize the molecular aspects of clinical gene transfer and review recent results and future prospects of gene therapy applications for immunodeficiency diseases.