NEURONAL CELL THERAPY FOR PARKINSONS DISEASE

hNT-神经元的独特性质，纯人类有丝分裂后神经元 细胞，证明他们的发展作为一个本地交付系统， 多巴胺能因子对帕金森病的治疗益处。 以前的研究表明，hNT神经元是可移植的， 可转染的 hNT-神经元在啮齿动物脑中的移植显示细胞 存活、神经元表型和功能整合。 转染研究表明，重组hNT-神经元可以实现 高效稳定的基因表达。 这项建议的目的是 评价hNT-神经元的内源性多巴胺能特性; 用酶、辅因子或生长增强多巴胺能活性 调查额外的克隆和亚克隆， 生产;并确定最佳的基因构建体进行筛选， 治疗目的。 酶的表达和L-多巴的生产将 使用免疫化学和生物化学测定来测量。 这 研究策略允许表征和优化 hNT-神经元在体外的多巴胺能特性 II期计划的研究。 功能性，多巴胺能，人神经元 细胞在科学上是有前途的，在技术上比其他细胞优越上级， 细胞系统，并且作为帕金森氏症的治疗方法在商业上可行 疾病

The unique properties of hNT-Neurons, pure human post mitotic neuronal cells, justify their development as a local delivery system for dopaminergic factors of therapeutic benefit in Parkinson's disease. Previous research demonstrates that hNT-Neurons are transplantable and transfectable. Transplants of hNT-neurons into rodent brain show cell survival, neuronal phenotype, and functional integration. Transfection studies suggest that recombinant hNT-neurons can achieve efficient and stable gene expression. The aims of this proposal are to: evaluate the endogenous dopaminergic properties of hNT-Neurons; enhance dopaminergic activity with enzymes, cofactors or growth factors; survey additional clones and subclones for highest production; and identify the optimal gene construct to transfect for therapeutic purposes. Enzyme expression and L-dopa production will be measured using immunochemical and biochemical assays. This research strategy allows for characterization and optimization of the dopaminergic properties of hNT-Neurons in vitro prior to animal studies planned in Phase II. Functional, dopaminergic, human neuronal cells are scientifically promising, technologically superior to other cell systems, and commercially viable as a treatment for Parkinson's disease.