Grant Award Details
MSC-delivered artificial transcription factor to the brain as a treatment for Angelman Syndrome
Grant Application Details
- MSC delivery of an artificial transcription factor to the brain as a treatment for Angelman Syndrome
Mesenchymal stem cells will be used to deliver an artificial transcription factor to neurons in the brain to treat a genetic disease.
It could lead directly to a treatment for Angelman Syndrome, but the approach could be used to alter gene expression in almost any brain disorder. It could overcome the brain delivery bottleneck.
Major Proposed Activities
- Prepare the MSC delivery system (month 1 – month 6)
- Rescue and analysis of on-target molecular phenotypes in “YFP-mice” (month 6 – month12)
- Rescue and analysis of the behavioral phenotypes in “AS-mice” (month 12 – month 24)
- Analysis of the off-target molecular phenotypes in “YFP-mice” (month 18 – month 24)
Brain disorders are responsible for more years lost to disability than any other medical condition. For example, autism spectrum disorder (ASD) in the US is estimated to affect 1 in 68 children. The need for effective treatments can not be understated. Molecular therapeutics pioneered to understand and treat rare single-gene disorders such as Angelman Syndrome will provide the tools and methods that will ultimately be used to address the more common complex brain disorders.