New England Journal of Medicine
August 1, 2019 Vol. 381 No. 5
Frontiers in Medicine: Gene Therapy
Katherine A. High, M.D., and Maria G. Roncarolo, M.D.
Those who have followed the gene-therapy field over the decades may be weary of forward-looking positive statements. However, over the past 3 years, six gene-therapy products have been approved for clinical use. This article describes challenges, risks, and advances in gene-therapy clinical research.
Interview with Dr. Katherine A. High on gene therapy for genetic disease. (15:22) Download