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A Translational Pathway Toward a Clinical Trial Using the Second-Generation AAV Micro-Dystrophin Vector

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Technical Report,01 Sep 2014,31 Aug 2015

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University of Missouri System Columbia United States

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Duchenne muscular dystrophy DMD is a life threatening disease affecting all muscles in the body. An important therapeutic goal of DMD gene therapy is to deliver a therapeutic gene to all muscles in the body. The overarching goal of this project is to achieve systemic AAV-8 mediated expression of a low immunogenic human micro-dystrophin gene to young adult affected dogs. In this funding period, we demonstrated that we can achieve efficient whole body skeletal muscle and heart gene transfer in neonatal dogs with AAV-8 by a single intravenous injection. We also showed that systemic delivery of a caninemicro-dystrophin AAV vector is safe in young adult affected dogs. These results established a solid foundation to test systemic low-immunogenic human microgene delivery in young adult affected dogs in subsequent years of this project. In addition, we have performed a comprehensive review on the current status of DMD gene therapy in the canine model. We also contributed another article and reviewed the use of animal models in the development of DMD gene therapy.

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