Immune cloaking of human stem cell-derived insulin-producing cells for curative cell therapy without immunosuppression
Grant Award Details
Grant Type:
Grant Number:
DISC0-15972
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Cell Line Generation:
Award Value:
$1,192,586
Status:
Pre-Active
Grant Application Details
Application Title:
Immune cloaking of human stem cell-derived insulin-producing cells for curative cell therapy without immunosuppression
Public Abstract:
Research Objective
The goal of our project is to generate cells for replacement therapy in patients that have reduced ability to trigger the immune response in the recipient and therefore escape rejection.
Impact
We test a novel molecule that reduces immune activation upon transplantation of allogeneic stem cell products that can improve graft survival and as a result alleviate disease symptoms.
Major Proposed Activities
The goal of our project is to generate cells for replacement therapy in patients that have reduced ability to trigger the immune response in the recipient and therefore escape rejection.
Impact
We test a novel molecule that reduces immune activation upon transplantation of allogeneic stem cell products that can improve graft survival and as a result alleviate disease symptoms.
Major Proposed Activities
- Remove a DNA modifying factor from human pluripotent stem cells and test if the edited cells can differentiate into functional insulin-producing cells
- Test the immune-resilience of edited human cells when combined with immune cells in culture as well as in mouse models.
Statement of Benefit to California:
Diabetes is largely a patient-managed disease, and health literacy has a strong correlation with glycemic control. Underserved communities suffer disproportionately from complications, further reducing their quality of life. Our intention is to reach all patients with insulin-dependent diabetes, many of whom are Spanish-speaking California residents. Making therapies accessible to patients who face daily challenges of living with diabetes addresses a clear need in California communities.