Somatic Gene Therapy: Ethics and Access.

Manipulation of a patient's genome for therapeutic ends is being attempted through numerous methods, some of which have resulted in disease-modifying interventions. The much anticipated promise of somatic gene therapy is starting to pay off; however, there remain many scientific unknowns, including concerns about safety and durability. A significant ethical concern is that of access to these novel interventions, an issue that is normally framed in terms of the high costs of approved products. I describe how access issues permeate gene therapy long before there is any commercial product and how even upstream decisions—such as choices of indication to pursue, viral vector, and where to site a trial—have significant implications for access to resultant products in both the developmental and commercial stages. [ABSTRACT FROM AUTHOR]