… muscle and heart throughout life To study the longitudinal effects of systemic AAV lDys gene
therapy… DISCUSSION In this study, we evaluated lifelong systemic AAV lDys gene therapy in …

… compared therapeutic efficacy of an AAV9 micro-dystrophin … ) system in a Duchenne
muscular dystrophy mouse model. … Micro-dystrophin gene therapy goes systemic in Duchenne …

… It appears that the acceleration of cardiac disease progression we observed following
high dose systemic delivery of AAV-ΔR3-R21ΔCT-micro-dystrophin is primarily due to loss of …

… Adeno-associated viral (AAV) vector delivery of an early generation micro-dystrophin
prevented cardiac pathology and functional decline through 1 year of age in this new model. We …

… DD is an inventor on related intellectual property owned by the University of Missouri:
Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma, US patent number …

… to test micro-dystrophin gene therapy efficacy on heart failure prevention for the first time.
Micro-dystrophin … micro-dystrophin (AAV-μDys) suggest great promise for improving skeletal …

… -of-principle for systemic gene transfer using AAV in humans [… different mini-/ micro-dystrophin
constructs delivered using … in the efficiency of systemic AAV transduction. For example, …

… optimizing dosage within the micro-dystrophin gene would increase dystrophin expression
… AAV in gene therapy, we chose carrying out transduction using LV treatment due to the AAV …

… at assessing the impact of the IM delivery of an AAV micro-dystrophin gene (rAAV2.5-CMV-mini-…
More recently, systemic AAV delivery of a transgene encoding micro-dystrophin was …

… the micro-dystrophin transgene sequence, the efficacy and safety of AAV micro-dystrophin
gene therapy are … Therefore, to maximize gene expression in cardiac and skeletal muscle, the …