Development of platforms for beta cell-specific delivery and ligand discovery
Contact PI: Amit Choudhary, PhD, Broad Institute (U01 DK137242)
Rohit Kulkarni, MD, PhD, Joslin Diabetes Center
Paul Andrew Clemons, PhD, Broad Institute
Start Date: July 1, 2023
Patients suffering from type 1 diabetes must undergo burdensome, often lifelong, exogenous insulin dependence. Up to now, the only available replacement therapy is to transplant islets from cadaveric donors. However, such procedures present hurdles such as the scarcity of available donors and the rejection of transplanted cells by the patient’s own immune system. Also, over time, the transplanted islets tend to die. To circumvent these effects, we and other have identified several therapeutic targets to induce beta cell proliferation. However, the realization of the therapeutic potential of these targets requires targeted release as the therapeutic window of these targets is narrow. We propose to apply chemical biology approaches to develop methods for targeted release of bioactives in beta cells in vivo.