OPTIMIZE HUMAN NEURONAL CELLS FOR SPINAL CORD TRANSPLANT

描述（由申请人提供）：hNT-神经元是纯人类神经元细胞 其表现出神经元表型，并在 移植到啮齿动物脊髓中。第一阶段研究成功开展 具有适合于脊髓移植的特性的hNT-神经元， 确定了最大限度地提高移植成功率的关键实验条件， 体内，并证明移植物存活在啮齿动物模型的临床相关 颈脊髓挫伤第二阶段的研究将集中在进一步 产品开发，优化嫁接方案， 长期安全性的解剖学、神经生理学和行为学研究， 功效具体目的包括：（1）检测移植物存活率、细胞增殖率、 分化、神经递质表型谱和神经炎生长 作为移植的hNT-神经元的细胞密度的函数;和（2）建立 通过监测安全性特征来评估长期安全性和功能有效性， 膈运动神经元功能和前肢运动行为的长期变化 脊髓挫伤中hNT-神经元移植后的时间。的 这些实验的结果将有助于确定 hNT神经元改善脊髓损伤的结果，并提供有价值的 必要的信息，以推进临床试验。 拟定商业应用： 不可用

DESCRIPTION (provided by applicant): hNT-Neurons are pure human neuronal cells that exhibit neuronal phenotypes and efficiently engraft following transplantation into rodent spinal cord. Phase I studies successfully developed hNT-Neurons with properties suitable for spinal cord transplantation, identified the critical experimental conditions to maximize graft success in vivo, and demonstrated graft survival in a rodent model of clinically relevant cervical spinal cord contusion injury. Phase II studies will focus on further product development, optimization of grafting protocols, and intensive anatomical, neurophysiological and behavioral studies of long-term safety and efficacy. Specific aims include: (1) examine graft survival, rate of cellular differentiation, neurotransmitter phenotypic profiles, and neuritic outgrowth as a function of the cell density of grafted hNT-Neurons; and (2) establish long-term safety and functional efficacy by monitoring the safety profile, phrenic motoneuron function, and forelimb motor behavior over extended time periods following grafts of hNT-Neurons in spinal cord contusion injury. The results of these experiments will help establish the therapeutic potential of hNT-Neurons to improve outcomes in spinal cord injury and provide valuable information necessary to move ahead toward clinical trials. PROPOSED COMMERCIAL APPLICATION: Not Available