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The Michael J. Fox Foundation’s Strategies for Accelerating Translation of LRRK2 into Therapies for Parkinson Disease

机译:Michael J. Fox基金会加速LRRK2翻译成帕金森病的疗法的策略

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摘要

Since 2005, The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has invested significant funding and non-funding effort to accelerate research and drug development activity around the Parkinson disease (PD)-associated protein LRRK2. MJFF has spearheaded multiple public/private pre-competitive collaborations that have contributed to our understanding of LRRK2 function; de-risked potential safety questions around the therapeutic use of LRRK2 kinase inhibitors; and generated critical research tools, biosamples, and data for the field. Several LRRK2-targeted therapies are now in human testing due to the hard work of so many in the PD community. In this perspective, we present a holistic description and model of how our Foundation’s support targeted important barriers to LRRK2 research and helped move the field into clinical trials.
机译:自2005年以来,迈克尔J.Fox为帕金森的研究(MJFF)投资了大量资金和非资金努力,以加速帕金森病(Pd)蛋白LRRK2周围的研究和药物开发活动。 MJFF已经为我们对LRRK2功能的理解做出了贡献的多个公众/私人前竞争力的合作;在治疗使用LRRK2激酶抑制剂的情况下失败的潜在安全问题;并生成关键的研究工具,生物素质和数据的数据。由于PD社区中如此多的努力,几个LRRK2目标疗法现在正在人体测试中。在这种观点中,我们提供了一个整体描述和模型,我们的基金会支持如何对LRRK2研究的重要障碍,并帮助将现场移入临床试验中。

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