Designing a cellular niche for transplantation of human embryonic stem cell-derived beta cells

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Grant Award Details

Grant Number:
DISC2-09635
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$2,006,076
Status:
Closed

Progress Reports

Reporting Period:
Year 2
Reporting Period:
NCE

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.