Grant Award Details

MSC delivery of an artificial transcription factor to the brain as a treatment for Angelman Syndrome
Grant Number: 
DISC2-09032
Project Objective: 
  • MSC-delivered artificial transcription factor to the brain as a treatment for Angelman Syndrome

Investigator: 
Type: 
PI
Disease Focus: 
Autism
Neurological Disorders
Human Stem Cell Use: 
Adult Stem Cell
Award Value: 
$1,087,572
Status: 
Active

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

  • 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.