2008

Gene Therapy Restores Partial Sight — Some 20 years after the idea first came to them, Jean Bennett, M.D., Ph.D., and Albert M. Maguire, M.D., have used gene therapy to restore partial sight to patients with Leber's congenital amaurosis (LCA). A severe form of retinal degradation, the condition leads to total blindness. The preliminary results set the stage for further studies of an innovative treatment for LCA and possibly other retinal diseases.

The F.D.A. has referred to the "New Era of Personalized Medicine" and last year supported it with $277 million. Some private groups see its enormous potential for making money. Many physicians are enthusiastic about its clinical impact. Although some questions about cost and privacy remain, the movement toward personalized medicine is very strong. At PENN Medicine, cancer specialists are among those taking the lead in this new paradigm.