Gene therapy for pompe disease effective in mice, poised for human trials – EurekAlert (press release)
EurekAlert (press release) |
Gene therapy for pompe disease effective in mice, poised for human trials
EurekAlert (press release) The Duke-led research team found that a single small dose of gene therapy was as effective as enzyme replacement therapy (ERT) in clearing the buildup of glycogen from the muscles in mice. A larger dose offered superior results to ERT. A single … |
