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

1型糖尿病患者必须承受沉重的，通常是终生的外源性胰岛素依赖。到目前为止，唯一可行的替代疗法是从尸体捐献者身上移植胰岛。然而，这种方法存在一些障碍，如供体稀缺和患者自身免疫系统对移植细胞的排斥。此外，随着时间的推移，移植的胰岛往往会死亡。为了避免这些影响，我们和其他人已经确定了几个治疗靶点来诱导β细胞增殖。然而，这些靶点的治疗潜力的实现需要靶向释放，因为这些靶点的治疗窗口很窄。我们建议应用化学生物学方法来开发生物活性物质在体内β细胞中的靶向释放方法。

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