Development of platforms for beta cell-specific delivery and ligand discovery

Broad Institute, Inc., Cambridge MA

Investigators

Abstract

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. 1