The studies will identify and characterize new gene therapy vectors able to deliver gene editing components to stem cells. to enable treatment of diseases involving both muscle and brain.
New AAV capsids that target muscle and neural stem cells will enhance the number of neurological diseases able to be treated with AAV-based approaches
Major Proposed Activities
The project has the potential to create a valuable resource that could be used in research or to treat patients with devastating neurological disorders. The project will employ four individuals to carry out the project, which will create jobs within the state.