MSC delivery of an artificial transcription factor to the brain as a treatment for Angelman Syndrome
Grant Award Details
Grant Type:
Grant Number:
DISC2-09032
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$1,055,319
Status:
Closed
Progress Reports
Reporting Period:
Year 2/NCE
Grant Application Details
Application Title:
MSC delivery of an artificial transcription factor to the brain as a treatment for Angelman Syndrome
Public Abstract:
Research Objective
Mesenchymal stem cells will be used to deliver an artificial transcription factor to neurons in the brain to treat a genetic disease.
Impact
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
Mesenchymal stem cells will be used to deliver an artificial transcription factor to neurons in the brain to treat a genetic disease.
Impact
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)
Statement of Benefit to California:
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.
Publications
- Front Mol Neurosci (2021): An in vivo Cell-Based Delivery Platform for Zinc Finger Artificial Transcription Factors in Pre-clinical Animal Models. (PubMed: 35153670)
