Development of Novel Micro-dystrophins with Enhanced Functionality

著者: Julian N. Ramos, Katrin Hollinger, Niclas E. Bengtsson, J. M. Allen, Stephen D. Hauschka, Jeffrey S. Chamberlain - Molecular Therapy 2019 被引用: 114

mice, followed by analysis of skeletal muscle pathophysiology. Two μDys designs were identified that led to increased force generation compared with previous μDys while also localizing neuronal nitric oxide synthase to the sarcolemma. An AAV vector expressing the smaller of these (μDys5) from the CK8e RC is currently being evaluated in a DMD clinical trial.