一种针对PRKN/帕金蛋白的分子胶。
A molecular glue for PRKN/parkin.
作者信息
Sauvé Véronique, Gehring Kalle
机构信息
Department of Biochemistry and Centre de Recherche en Biologie Structurale, McGill University, Montreal, QC, Canada.
出版信息
Autophagy. 2025 Mar;21(3):689-690. doi: 10.1080/15548627.2024.2443232. Epub 2024 Dec 24.
Parkinson disease (PD) is a neurodegenerative disease characterized by the loss of dopaminergic neurons in the , primarily due to mitochondria dysfunction. PRKN (parkin RBR E3 ubiquitin protein ligase) and PINK1 (PTEN induced kinase 1) are linked to early-onset cases of PD and essential for the clearance of damaged mitochondria via selective mitochondrial autophagy (mitophagy). In a recent publication, we detail how a small molecule can activate PRKN mutants that are unable to be phosphorylated, restoring mitophagy in cellular assays. These findings offer hope for the design of therapeutic drugs for some forms of PD.
帕金森病（PD）是一种神经退行性疾病，其特征是黑质中多巴胺能神经元的丧失，主要原因是线粒体功能障碍。PRKN（帕金RBR E3泛素蛋白连接酶）和PINK1（PTEN诱导激酶1）与早发性帕金森病病例相关，并且对于通过选择性线粒体自噬（线粒体自噬）清除受损线粒体至关重要。在最近的一篇出版物中，我们详细阐述了一种小分子如何激活无法被磷酸化的PRKN突变体，在细胞试验中恢复线粒体自噬。这些发现为某些形式的帕金森病治疗药物的设计带来了希望。
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