Engineering AAV capsids for transduction of neural and muscle stem cells

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Grant Award Details

Grant Number:
DISC0-13765
Investigator(s):
Human Stem Cell Use:
Award Value:
$999,999
Status:
Active

Grant Application Details

Application Title:

Engineering AAV capsids for transduction of neural and muscle stem cells

Public Abstract:
Research Objective

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.

Impact

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

  • Screen an AAV library to identify AAV capsids that transduce both muscle and neural stem cells.
  • Test the best capsids in human cells and tissues.
Statement of Benefit to California:
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.