A molecular glue for PRKN/parkin.

Véronique Sauvé, Kalle Gehring
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Abstract

Parkinson disease (PD) is a neurodegenerative disease characterized by the loss of dopaminergic neurons in the substantia nigra, 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.

用于Prkn/parkin的分子胶。
帕金森病(PD)是一种神经退行性疾病,其特征是黑质多巴胺能神经元的丧失,主要是由于线粒体功能障碍。PRKN (parkin RBR E3泛素蛋白连接酶)和PINK1 (PTEN诱导的激酶1)与早发性PD病例有关,并且通过选择性线粒体自噬(mitophagy)清除受损线粒体至关重要。在最近发表的一篇文章中,我们详细介绍了一个小分子如何激活无法磷酸化的PRKN突变体,在细胞分析中恢复有丝分裂。这些发现为设计治疗某些形式帕金森病的药物提供了希望。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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