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首页> 美国卫生研究院文献>Biomolecules >The Michael J. Fox Foundation for Parkinson’s Research Strategy to Advance Therapeutic Development of PINK1 and Parkin
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The Michael J. Fox Foundation for Parkinson’s Research Strategy to Advance Therapeutic Development of PINK1 and Parkin

机译：迈克尔·J·福克斯基金会（Michael J. Fox Foundation）为帕金森氏症（PINK1和Parkin）的治疗性发展研究战略

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The role of mitochondria in Parkinson’s disease (PD) has been investigated since the 1980s and is gaining attention with recent advances in PD genetics research. Mutations in PRKN and PTEN-Induced Putative Kinase 1 (PINK1) are well-established causes of autosomal recessive early-onset PD. Genetic and biochemical studies have revealed that PINK1 and Parkin proteins function together in the same biological pathway to govern mitochondrial quality control. These proteins have also been implicated in the regulation of innate and adaptive immunity and other mitochondrial functions. Additionally, structural studies on Parkin have delineated an activation mechanism and have identified druggable regions that are currently being explored by academic and industry groups. To de-risk therapeutic development for these genetic targets, The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has deployed a strategic funding and enabling framework that brings together the research community to discuss important breakthroughs and challenges in research on PINK1-Parkin biology, supports collaborative initiatives to further our understanding within this field and develops high-quality research tools and assays that are widely available to all researchers. The Foundation’s efforts are leading to significant advances in understanding of the underlying biology of these genes, proteins and pathways and in the development of Parkinson’s therapies.
机译：自1980年代以来，线粒体在帕金森氏病（PD）中的作用得到了研究，并且随着PD遗传学研究的最新进展而引起人们的关注。 PRKN和PTEN诱导的假定激酶1（PINK1）中的突变是常染色体隐性遗传性早发性PD的公认原因。遗传和生化研究表明，PINK1和Parkin蛋白在同一生物学途径中共同起作用，以控制线粒体质量控制。这些蛋白质还涉及先天和适应性免疫以及其他线粒体功能的调节。此外，对帕金森氏症的结构研究已勾画出一种激活机制，并确定了目前学术界和工业界正在探索的可药物化区域。为了降低这些基因靶标的治疗发展风险，迈克尔·J·福克斯基金会（Parkingson's Research）的迈克尔·J·福克斯基金会（MJFF）部署了战略资金和支持框架，该框架将研究界聚集在一起，讨论PINK1-Parkin生物学研究的重要突破和挑战支持各种合作计划，以加深我们对该领域的了解，并开发可广泛用于所有研究人员的高质量研究工具和测定法。基金会的努力在理解这些基因，蛋白质和途径的基本生物学以及开发帕金森疗法方面取得了重大进展。

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