Human Gene TherapyA developing treatment that aims to fix, replace, or add genetic instructions. 9:1609-1616 (July 20, 1998) Mary Ann Liebert, Inc. Adenovirus-Mediated Transfer of Human Acid Maltase Gene Reduces Glycogen

National Institutes of Health Paul Plotz, M.D., Nina Raben, M.D., Ph.D. Nina Raben, M.D., Ph.D., NIH Research Chemist, visited the

Human Molecular Genetics, January 1998 “Generalized glycogenA stored form of sugar used for energy. storage and cardiomegaly in a knockout mouse modelA laboratory mouse used to study disease and test treatments before human trials. of Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart.” by A.

Johns Hopkins University Barry J. Byrne, M.D., Ph.D. Paul D. Kessler, M.D. The following is a summary of the publication

Johns Hopkin’s University scientists have successfully used a virus to supply a missing gene and its enzymeA protein that helps the body carry out chemical reactions. product to muscle