Basic science and preclinical development of cell transplantation for basal ganglia disorders

该计划提供基础设施、模型和专业知识，以提供用于神经退行性疾病细胞移植临床试验的人类神经元（原代胚胎、扩增胚胎和干细胞）。具体来说，在卡迪夫脑修复小组，我们已经建立了一个道德来源的供应和质量控制的人类胚胎脑细胞的生产，用于亨廷顿？s和帕金森？年代的疾病。我们还建立了细胞培养和动物模型测试系统，以确定最有可能在PD和HD的2011-12年开始/恢复的试验中带来功能益处（即运动和认知症状的恢复）的精确制备和植入方案。此外，英国在基础干细胞生物学方面处于世界领先地位，这可能会产生用于治疗各种疾病的下一代多能细胞；然而，如果我们要从政府在这一领域的大力支持和公共投资中获益，我们需要有基础设施来测试新的干细胞制剂的功能功效，使细胞达到临床级质量标准，并在特征明确的患者中进行熟练的手术和充分验证的评估方案的临床试验。因此，除了目前建立的原代胎儿细胞概念验证外，该计划拨款还支持卡迪夫脑修复小组的专业知识和基础设施，这些专业知识和基础设施需要在新的干细胞疗法可用后尽快将其投入临床试验。如果没有这种积极的准备和支持，英国在基础干细胞生物学方面的智力领先地位很可能被浪费在其他地方的实施和商业化上。

This programme provides the infrastructure, models and expertise to deliver human neurons (primary fetal, expanded fetal and stem cells) for use in clinical trials of cell transplantation in neurodegenerative disease. Specifically, in the Cardiff Brain Repair Group, we have established an ethically sourced supply and quality-controlled production of human embryonic brain cells for transplantation in both Huntington?s and Parkinson?s disease. We have also established the cell culture and animal model test systems to determine the precise preparation and implantation protocols that are most likely to lead to functional benefit (i.e. recovery in both motor and cognitive symptoms) in trials set to commence/resume in both PD and HD in 2011-12. Moreover, the UK is a world leader in fundamental stem cell biology which are likely to yield the next generation of pluripotential cells for therapy in a wide range of diseases; however, if we are to reap the benefit from the signficant government support and public investment in this area, we need to have in place the infrastructure to test the functional efficacy of new stem cell-based preparations, to prepare cells to clinical-grade quality standards, and to undertake clinical trials with skilled surgery and fully validated assessment protocols in well characterised patients. Thus, alongside the present establishment of proof-of concept with primary fetal cells, this programme grant underpins the expertise and infrastructure within the Cardiff Brain Repair Group that is required to take new stem cell therapies rapidly into clinical trial as soon as they become available. Without such proactive preparation and support, the UKs intellectual lead in fundamental stem cell biology is likely to be squandered to implementation and commercialisation elsewhere.