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Introduction: Gene and Cell Therapy Projects of the Seventh Framework Program

机译:简介:第七框架计划的基因和细胞疗法项目

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We present herein the first series of 10 recently finished or ongoing gene therapy projects funded by the European Union during the seventh framework program for research and innovation (2007-2013). The majority of these projects address rare diseases (AIPgene, Genegraft, Cell-PID, OptiStem, Treatrush, PERSIST), while some investigate common disorders such as cancer (LeukoTreat), vascular (Biomagscar), bone (Gamba), or neurological disorders (BrainCAV). These international collaborative projects are multidisciplinary and gather the necessary expertise to progress from bench to bedside. The presentation of each of these projects includes main findings and publications in addition to their dedicated website where details about consortium's structure and achievements can be found. Throughout the years, our funding was targeted to support technological scale-up, good manufacturing practice, development of new and adequate preclinical models to foster new toxicology, and monitoring tests so that the technology could reach the patients. The support to the very innovative clinical trials required to challenge the European ethics and currently evolving regulatory context.
机译:我们在此介绍由欧盟在第七个研究与创新框架计划(2007-2013年)中资助的,最近完成或正在进行的10个基因治疗项目的第一个系列。这些项目中的大多数都针对罕见疾病(AIPgene,Genegraft,Cell-PID,OptiStem,Treatrush,PERSIST),而另一些项目则研究了常见的疾病,例如癌症(LeukoTreat),血管(Biomagscar),骨骼(Gamba)或神经系统疾病( BrainCAV)。这些国际合作项目是多学科的,并且聚集了必要的专业知识,可以从工作台发展到床边。这些项目的介绍除主要网站外,还包括主要发现和出版物,在该网站上可以找到有关财团的结构和成就的详细信息。多年来,我们的资金主要用于支持技术扩大规模,良好的生产规范,开发新的适当的临床前模型以培养新的毒理学以及监测测试,以便该技术可以覆盖患者。挑战欧洲道德规范和当前不断发展的监管环境所需的创新性临床试验的支持。

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