GENE THERAPY FOR DIABETES

糖尿病基因治疗

• 批准号: 7957906
• 负责人: LAWRENCE CHAN
• 金额: $ 9.44万
• 依托单位: TEXAS BIOMEDICAL RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-06-06 至 2010-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7957906
• 关键词: Computer Retrieval of Information on Scientific Projects Database; Dependence; Diabetes Mellitus; Freedom; Funding; Future; Genes; Grant; Human; Institution; Insulin; Liver; Papio; Physiology; Primates; Protocols documentation; Research; Research Personnel; Resources; Source; Toxic effect; Treatment Efficacy; United States National Institutes of Health; Virus; diabetic; gene therapy; human subject; islet; response

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.

该副本是利用众多研究子项目之一 由NIH/NCRR资助的中心赠款提供的资源。子弹和 调查员（PI）可能已经从其他NIH来源获得了主要资金， 因此可以在其他清晰的条目中代表。列出的机构是 对于中心，这不一定是调查员的机构。 该项目的最终目的是制定一种基因治疗方案，该方案将安全用于人类，并将逆转糖尿病，并消除或显着降低对胰岛素治疗糖尿病的依赖性。该项目将使用狒狒，因为它们最类似于人类的生理学，并最好地复制将来在人类受试者中会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，并评估其在治疗的糖尿病患者肝脏中诱导新的胰岛素产生胰岛形成的新胰岛素胰岛形成方面的治疗疗效。最后，我们将根据免受胰岛素需求的自由，从肝脏中产生的新胰岛产生胰岛素的能力以及该反应的持续时间进行比较。