SBIR Phase I: Neural Plasticity of Bone Marrow Derived Progenitor Cells

这个小企业创新研究第一阶段项目旨在开发一种使用成人干细胞治疗中枢神经系统疾病的新型细胞疗法。成人骨髓中的一种特殊干细胞(CD34“LIN”)已经被分离出来，它可以被诱导表达一种神经元表型，并特异性地定位于大脑。该项目假设，这些细胞的多潜能性质将使细胞替代和药物输送疗法能够治疗神经系统疾病或损伤，包括帕金森氏症和中风。第一阶段项目将研究CD34“Lin”细胞的分离和鉴定、体外分化和体内分化。该项目的商业应用是在人类医疗保健领域。这项技术可能会为帕金森氏症、中风和阿尔茨海默病等毁灭性疾病带来潜在的治疗方法。这些疾病中的每一种都代表着大量的患者，因此，这是一个巨大的市场机会。此外，尤其是对于中风、帕金森氏症和阿尔茨海默氏症等疾病，目前几乎没有替代策略可用。其他细胞疗法正在开发中，但这些疗法大多来自胎儿组织或异种来源，这会带来安全或伦理问题。

This Small Business Innovation Research Phase I project aims to develop a novel cell therapy using adult stem cells to treat diseases of the central nervous system. A specific stem cell (CD34 "Lin") within adult bone marrow has been isolated that can be induced to express a neuronal phenotype and specifically home to the brain. This project hypothesizes that the multipotential nature of these cells will enable cell replacement and drug delivery therapies to treat diseases or injury of the nervous system, including Parkinson's and stroke. The Phase I project will address isolation and characterization, in vitro differentiation, and in vivo differentiation of CD34 "Lin" cells. The commercial application of this project is in human healthcare. The technology could lead to potential therapeutic treatments for devastating diseases such as Parkinson's disease, stroke and Alzheimer's disease. Each of these diseases represents a substantial number of patients and therefore, a substantial market opportunity. Furthermore, particularly for diseases such as stroke, Parkinson's and Alzheimer's, there are few, if any alternative strategies currently available. Other cell therapies are being developed, but most of these are derived from fetal tissue or xenogeneic sources, which introduce safety or ethical concerns.