GENE THERAPY TO REVERSE DIABETES

逆转糖尿病的基因疗法

• 批准号: 8357703
• 负责人: Paul A Grayburn
• 金额: $ 3.07万
• 依托单位: TEXAS BIOMEDICAL RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-05-01 至 2012-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8357703
• 关键词: Acclimatization; Animals; Beta Cell; Development; Diabetes Mellitus; Funding; Genes; Glucose; Grant; Hyperglycemia; Injection of therapeutic agent; Insulin; Insulin-Dependent Diabetes Mellitus; Modeling; Monitor; National Center for Research Resources; Pancreas; Papio; Phase; Primates; Principal Investigator; Production; Recovery; Research; Research Infrastructure; Resources; Source; Streptozocin; Technology; Testing; Tissues; Ultrasonography; United States National Institutes of Health; blood glucose regulation; cost; gene therapy; uptake

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. Primary support for the subproject and the subproject's principal investigator may have been provided by other sources, including other NIH sources. The Total Cost listed for the subproject likely represents the estimated amount of Center infrastructure utilized by the subproject, not direct funding provided by the NCRR grant to the subproject or subproject staff. The primary aim of this study is the establishment of a model of induced type 1 diabetes in baboons through beta cell compromise with streptozotocin (STZ), with the subsequent recovery of insulin production through gene therapy by the delivery of a specific gene using micro-bubble technology and their subsequent selective destruction in the pancreas via ultrasound. This study will be conducted in six phases. . The first two phases will include animal selection and acclimation, followed by the induction of type 1 diabetes via STZ administration. Animals will then be allowed to recover and the development of hyperglycemia will be tracked. Once the animals are stabilized and hyperglycemia has been achieved the animals will receive an injection of the micro-bubbles containing the test gene followed by the destruction of the micro-bubbles in the pancreas via ultrasound. Animals will continue to have their glucose levels monitored at regular intervals to look for improvement in glucose regulation. At the end of the experimental period the animals will be euthanized and key tissues will be collected for histopathological analyses to confirm gene uptake in the pancreas.

这个子项目是许多利用资源的研究子项目之一 由NIH/NCRR资助的中心拨款提供。子项目的主要支持 而子项目的主要调查员可能是由其他来源提供的， 包括其它NIH来源。 列出的子项目总成本可能 代表子项目使用的中心基础设施的估计数量， 而不是由NCRR赠款提供给子项目或子项目工作人员的直接资金。 本研究的主要目的是通过β细胞妥协与链脲佐菌素（STZ）诱导狒狒1型糖尿病模型的建立，随后通过基因治疗恢复胰岛素生产，通过使用微泡技术递送特定基因，随后通过超声选择性破坏胰腺。 这项研究将分六个阶段进行。 . 前两个阶段将包括动物选择和驯化，然后通过STZ给药诱导1型糖尿病。 然后允许动物恢复，并跟踪高血糖症的发展。一旦动物稳定并且已经达到高血糖症，动物将接受含有测试基因的微泡的注射，然后通过超声破坏胰腺中的微泡。 将继续定期监测动物的葡萄糖水平，以寻找葡萄糖调节的改善。 在实验期结束时，将对动物实施安乐死，并采集关键组织进行组织病理学分析，以确认胰腺中的基因摄取。