Human gene therapy: a role for the primary care physician

Human gene therapy, once thought to be the unique province of specialized clinical centers, will be diffusing rapidly into primary care medicine. More than 10 medical centers in the United States and several centers world-wide are beginning to use this potentially curative therapy. Eleven trials of protocols are under way, nine are about to begin, and more than 12 protocols are nearing completion of the approval process. The diseases being treated are not the rare disorders found only in one in 100,000 patients, but instead include various types of cancer, diseases of the cardiovascular and pulmonary systems, and inborn errors of metabolism. Combined, these diseases affect more than half of the American population.