GENE THERAPY FOR DIABETES

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。子项目和 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以在其他 CRISP 条目中表示。列出的机构是 对于中心来说，它不一定是研究者的机构。 该项目的最终目标是开发一种可安全用于人类并逆转糖尿病的基因治疗方案，并消除或显着减少对胰岛素治疗糖尿病的依赖。该项目将使用狒狒，因为它们与人类生理学最相似，并且可以最好地复制未来在人类受试者中看到的治疗功效和潜在毒性。它将比较含有人类基因的病毒的毒性、持续时间和表达水平，并评估它们在诱导接受治疗的糖尿病受试者肝脏中产生新胰岛素的胰岛形成方面的治疗效果。最后，我们将比较无需胰岛素需求、从肝脏中产生的新胰岛产生胰岛素的能力以及这种反应的持续时间方面的反应。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The ultimate objective of this project is to develop a gene therapy protocol that will be safe for use in humans and will reverse diabetes and either eliminate or significantly reduce the dependence on insulin for treatment of diabetes. This project will use baboons as they most closely resemble human physiology and would best replicate the therapeutic efficacy and potential toxicities that would be seen in human subjects in the future. It will compare the toxicity and duration and level of expression of viruses containing human genes and evaluate their therapeutic efficacy in inducing new insulin producing islet formation in the livers of the treated diabetic subjects. Finally, we will compare the response in terms of freedom from insulin requirement, ability to produce insulin from the new islets generated in the liver, and the duration of this response.