Grant Award Details

Develop iPSC-derived microglia to treat progranulin-deficient Frontotemporal Dementia
Grant Number: 
DISC2-11165
Investigator: 
Name: 
Type: 
PI
Disease Focus: 
Dementia
Neurological Disorders
Human Stem Cell Use: 
iPS Cell
Award Value: 
$1,553,923
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • Develop iPSC-derived microglia to treat progranulin-deficient Frontotemporal Dementia
Public Abstract: 

Research Objective

Develop stem cell-based therapy to treat dementia

Impact

There are no treatments for dementia. If successfully achieved, this study will lead to a cure of a familial
 form of dementia in the elderly population.

Major Proposed Activities

  • Develop a robust human stem cell-derived microglial platform for cell-based therapy
  • Determine short-term safety and efficacy of engrafted human microglia in wildtype mice
  • Determine short-term efficacy of engrafted human microglia in FTD mouse models
  • Determine long-term efficacy of engrafted human microglia in FTD mouse models
Statement of Benefit to California: 

The proposed research will benefit the State of California and its citizens because of the potential to cure 
a major form of dementia in the elderly population. With the fast aging population in California, more and
 more Californians are diagnosed with neurodegenerative dementias. 
There is an urgent need to develop a treatment or cure for these devastating conditions. Success of our 
study will address this urgent medical challenge of our modern society.