Grant Award Details

Engineering AAV capsids for transduction of neural and muscle stem cells
Grant Type: 
Grant Number: 
DISC0-13765
Project Objective: 
  • The goal of the award is to identify recombinant AAV serotypes that are trophic for both muscle and neural stem cells- as the basis for future gene therapy efforts.
Investigator: 
Disease Focus: 
Skeletal/Smooth Muscle disorders
Human Stem Cell Use: 
iPS Cell
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.

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