GENE THERAPY DEVICE FOR TREATMENT OF HEMOPHILIA B

描述(未经编辑的申请人摘要)：本提案的目标是 开发一种用于治疗遗传性心脏病的新型基因治疗递送装置 无序。血友病B.一个毁灭性且代价高昂的市场(超过10亿美元) 以自发性内出血为特征的疾病。计划中的实验 将提供一种细胞治疗输送工具，用于植入大型 动物的器皿。最终，一名患有这种基因的患者 叛逃。特定目标1计划测试两个IVD原型制造 支持足够的成肌细胞数量和固定分泌率的能力 达到临床显著的固定水平。特定目标2计划优化 所选D/D原型的体外cFIX产生率。具体目标3 计划引入这种带有自体犬心肌细胞的IVD原型 将hFIX基因转导到正常狗体内，在那里它将固定在 绿地过滤器锚(GFA)的下腔静脉。它的血液相容性 下腔静脉内的装置，以及心肌细胞的活性将在 不同的时间段(最多6周)。特定目标4计划测试cFIX 转cFIX基因自体犬的产生率和生物学活性 血友病B犬模型IVD原型细胞中的成肌细胞。成功者 这些研究的完成将为使用 IVD作为一种基因疗法治疗血友病B的最终目标是 开发一种新的细胞和基因治疗递送系统(由 通过绿地过滤器锚定的单元格填充的IVD) 并且可通过经皮股静脉插入导管而缩回 进入下腔静脉。IVD可用于交付任何所需的 通过细胞或基因疗法合成化合物。这种方法可能是实现 技术，为基因治疗和细胞治疗行业提供了 安全的方法是植入可回收的设备直接引入基因产品 进入患有遗传性疾病的个体的体内循环。 建议的商业应用： 不可用

DESCRIPTION (Unedited Applicant's Abstract): The goal of this proposal is to develop a novel gene therapy delivery device for treatment of the genetic disorder. Hemophilia B. a devastating and costly (over a billion dollar market) disease characterized by spontaneous internal bleeding. The planned experiments will provide a cell therapy delivery vehicle for implantation into a large vessel of an animal. and eventually, a patient suffering from this genetic defect. Specific Aim 1 plans to test two IVD prototype fabrications for the ability to support adequate myoblast cell number and FIX secretion rates to achieve clinically significant FIX levels. Specific Aim 2 plans to optimize the in vitro cFIX production rates of the selected D/D prototype. Specific Aim 3 plans to introduce this IVD prototype with autologous canine myocytes transduced with hFIX gene into a normal dog where it will be secured in the vena cava by a Greenfield filter anchor (GFA). The hemocompatibility of this device within the vena cava, and myocytes viability will be assessed over various periods of time (up to 6 weeks). Specific Aim 4 plans to test the cFIX production rates and biological activity of cFIX transduced autologous canine myoblasts in the cell IVD prototype in a Hemophilia B dog model. The successful completion of these studies will provide 'proof of concept' for utilization of the IVD as a gene therapy' treatment of hemophilia B. The ultimate goal is to develop a novel cell and gene therapy delivery system (comprised of a cell-filled IVD anchored by means of a Greenfield filter) that is introducable and retractable via a percutaneous catheter insertion through the femoral vein into the inferior vena cava. The IVD may be used to deliver any desired compound via cell or gene therapy. This approach may be the key enabling technology for both the gene therapy and cell therapy industries, providing a safe means to implant a retrievable device to introduce gene products directly into the systemic circulation of an individual with a genetic disorder. PROPOSED COMMERCIAL APPLICATION: Not Available