Grant Award Details

Designing a cellular niche for transplantation of human embryonic stem cell-derived beta cells
Grant Number: 
DISC2-09635
Project Objective: 
  • The expected outcome of these studies is a cellular therapeutic for Type I Diabetes: engineered human islets for transplant into patients, surpassing the function of beta cells or progenitors alone.

Investigator: 
Disease Focus: 
Diabetes
Metabolic Disorders
Human Stem Cell Use: 
Embryonic Stem Cell
Award Value: 
$2,006,076
Status: 
Active

Progress Reports

Reporting Period: 
Year 2

Grant Application Details

Application Title: 
  • Designing a cellular niche for transplantation of human embryonic stem cell-derived beta cells
Public Abstract: 

Research Objective

The expected outcome of these studies is a cellular therapeutic for Type I Diabetes: engineered human islets for transplant
into patients, surpassing the function of beta cells or progenitors alone.

Impact

The proposed studies would address key bottlenecks in cell replacement therapy for Type I Diabetes -- issues with cellular engraftment, survival, and function -- enabling optimized delivery in vivo.

Major Proposed Activities

  • Determine the optimal composition of human embryonic stem cell (hESC)-derived engineered islets in vitro.
  • Define key pathways underlying the mechanisms of niche-induced maturation of hESC-derived beta-like cells.
  • Demonstrate function of engineered islets in vivo in immunodeficient animal models of type I diabetes.
Statement of Benefit to California: 

Type I Diabetes (T1D) is a significant burden in California, especially for children; according to estimates provided by the
California Diabetes Program, ~2.3 out of every 1,000 children between the ages of 5-19 in California had diagnosed diabetes
in 2008, with 83% having T1D. Research proposed here would represent a significant step towards the holy grail of T1D
treatment: a therapy for patients without the need for the administration of insulin, frequent blood testing, or
immunosuppression.