Genome editing of human Tregs to enable combinational tolerogenic therapy with T cell-targeted biologics for T1D
Grant Award Details
Grant Type:
Grant Number:
DISC2-16589
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$2,485,549
Status:
Active
Grant Application Details
Application Title:
Genome editing of human Tregs to enable combinational tolerogenic therapy with T cell-targeted biologics for T1D
Public Abstract:
Research Objective
Genome-edited autologous regulatory T cells for promoting immune tolerance in combination with T-cell-targeting therapies
Impact
We focus on type 1 diabetes in this proof-of-concept study, but the therapeutic platform the study will develop can be applied to many autoimmune indications and transplantation.
Major Proposed Activities
Genome-edited autologous regulatory T cells for promoting immune tolerance in combination with T-cell-targeting therapies
Impact
We focus on type 1 diabetes in this proof-of-concept study, but the therapeutic platform the study will develop can be applied to many autoimmune indications and transplantation.
Major Proposed Activities
- Develop genome editing protocol to modify human regulatory T cells
- Assess function of genome edited human Tregs in vitro
- Assess the efficacy of combinational therapy of genome-edited human Tregs with T cell-targeted immunotherapies in models of graft versus host disease in a humanized mouse model
- Establish a humanized mouse model of type 1 diabetes
- Develop genome editing protocol to modify mouse regulatory T cells from humanized mouse
- Assess the efficacy of combinational therapy of genome-edited mouse Tregs with T cell targeted immunotherapies in the humanized mouse models type 1 diabetes
Statement of Benefit to California:
Type 1 diabetes (T1D) affects 1.6 million in the US, including many Californians. Insulin is a life saving therapy for T1D by replacing this vital hormone that the patients cannot produce. A more effective way to treat T1D is to target the root cause of the disease - a faulty immune system. This project aims to develop an immune therapy by genetically modifying patients' own cells to restore immune self control. This therapeutic platform may also biotech industry in California.
