Human Gene Therapy 9:1609-1616
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 AccumulationThe buildup of glycogen inside cells and tissues when it is not broken down properly. in Skeletal MuscleMuscles that control movement and are commonly affected in Pompe disease. of Japanese Quail with Acid Maltase Deficiency S. Tsujino 1), N. Kinoshita 1), T. Tashiro 1),...
Gene Therapy: National Institutes of Health
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 Netherlands to collaborate with Dr. Arnold Reuser on genetic mutationsA change in a gene that can affect how it works. in AMD. They studied the presence of certain disease related...
Netherland’s Scientists Produce Knockout Mouse Model of Pompe disease
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. J. J. Reuser/A. T. van der Ploeg, et al Enzyme Replacement Therapy (ERT)A treatment that replaces the missing enzyme through IV infusion........... Therapy for Glycogen Storage Disease Type IIThe scientific name for Pompe disease, based on how glycogen builds up in the body. Acid...
Gene Therapy: Johns Hopkins University
Johns Hopkins University Barry J. Byrne, M.D., Ph.D. Paul D. Kessler, M.D. The following is a summary of the publication in the November 1996 issue of Proceeding of the National Academy of Science, titled “Gene delivery to skeletal muscle results in sustained...
Scientists Make Progress In Gene Therapy For Heart Disease
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 cells in animals and humans for an extended period. The achievement could have implications in the treatment of an inherited fatal heart...