GENE THERAPY FOR DIABETES

该子项目是利用 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 该项目的最终目标是开发一种基因治疗方案，该方案将安全用于人类，并将逆转糖尿病，消除或显著减少对胰岛素治疗糖尿病的依赖。该项目将使用狒狒，因为它们最接近人类生理学，并且最能复制未来在人类受试者中观察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，并评价它们在治疗的糖尿病受试者的肝脏中诱导产生新的胰岛素的胰岛形成的治疗功效。最后，我们将比较从胰岛素需求的自由度，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.