GENE THERAPY FOR DIABETES

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 该项目的最终目标是开发一种基因治疗方案，该方案将在人类中安全使用，并将逆转糖尿病，消除或显著减少治疗糖尿病对胰岛素的依赖。该项目将使用狒狒，因为它们最接近人类生理，并将最好地复制未来在人类受试者中看到的治疗效果和潜在毒性。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，并评估它们在治疗糖尿病受试者肝脏中诱导新的胰岛素分泌胰岛形成的治疗效果。最后，我们将从免除胰岛素需求、从肝脏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等方面比较这两种反应。

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.