Emergence of a scientific and commercial research and development infrastructure for human gene therapy

Abstract

A research and clinical subfield known as "human gene therapy" has grown rapidly since 1990, when the first human trials were approved in the United States. Using quantitative data, this paper describes and analyzes the research and commercial infrastructure, including academic centers, publications, intellectual property, and biotechnology firms, that has developed around the goal of discovering clinical applications for the modification and transport of DNA to somatic cells. Despite setbacks and few documented successes, the subfield of human gene therapy continues to serve as an influential clinical paradigm for the treatment of inherited and noninherited diseases.