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Reviews A Look to Future Directions in Gene Therapy Research for Monogenic Diseases * Matthew H. Porteus , Jon P. Connelly, Shondra M. Pruett ABSTRACT adeno-associated viruses, herpes simplex viruses, and retrovirus (including foamy and lentiviruses). A thorough he concept of gene therapy has long appealed to discussion of these vector delivery systems is beyond the Tbiomedical researchers and clinicians because it scope of this review, and comprehensive discussions of the promised to treat certain diseases at their origins. In different viral vectors used in gene therapy can be found the last several years, there have been several trials in which elsewhere [2–4]. patients have benefited from gene therapy protocols. This progress, however, has revealed important problems, Summary of Recent Clinical Trials of Gene Therapy including the problem of insertional oncogenesis. In this for Monogenic Diseases review, which focuses on monogenic diseases, we discuss the In the last decade there have been four monogenic diseases problem of insertional oncogenesis and identify areas for for which seminal gene therapy trials have been conducted: future research, such as developing more quantitative assays ornithine transcarbamylase (OTC) deficiency, hemophilia for risk and efficacy, and ways of minimizing the genotoxic