Instituto de Biomedicina de Valencia, CSIC and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Jaime Roig 11, 46010 Valencia, Spain.
Biochem J. 2011 Oct 15;439(2):265-75. doi: 10.1042/BJ20110150.
Lafora progressive myoclonus epilepsy [LD (Lafora disease)] is a fatal autosomal recessive neurodegenerative disorder caused by loss-of-function mutations in either the EPM2A gene, encoding the dual-specificity phosphatase laforin, or the EPM2B gene, encoding the E3-ubiquitin ligase malin. Previously, we and others showed that laforin and malin form a functional complex that regulates multiple aspects of glycogen metabolism, and that the interaction between laforin and malin is enhanced by conditions activating AMPK (AMP-activated protein kinase). In the present study, we demonstrate that laforin is a phosphoprotein, as indicated by two-dimensional electrophoresis, and we identify Ser(25) as the residue involved in this modification. We also show that Ser(25) is phosphorylated both in vitro and in vivo by AMPK. Lastly, we demonstrate that this residue plays a critical role for both the phosphatase activity and the ability of laforin to interact with itself and with previously established binding partners. The results of the present study suggest that phosphorylation of laforin-Ser(25) by AMPK provides a mechanism to modulate the interaction between laforin and malin. Regulation of this complex is necessary to maintain normal glycogen metabolism. Importantly, Ser(25) is mutated in some LD patients (S25P), and our results begin to elucidate the mechanism of disease in these patients.
拉佛拉病(Lafora 病)进行性肌阵挛癫痫是一种致命的常染色体隐性神经退行性疾病,由 EPM2A 基因(编码双重特异性磷酸酶 laforin)或 EPM2B 基因(编码 E3-泛素连接酶 malin)的功能丧失突变引起。以前,我们和其他人表明,laforin 和 malin 形成了一个功能复合物,调节糖原代谢的多个方面,并且 laforin 和 malin 之间的相互作用通过激活 AMPK(AMP 激活的蛋白激酶)的条件得到增强。在本研究中,我们证明 laforin 是一种磷酸化蛋白,二维电泳表明,我们确定 Ser(25)是参与这种修饰的残基。我们还表明,Ser(25)在体外和体内都被 AMPK 磷酸化。最后,我们证明该残基对于 laforin 的磷酸酶活性及其与自身和先前建立的结合伙伴相互作用的能力都至关重要。本研究的结果表明,AMPK 对 laforin-Ser(25)的磷酸化提供了一种调节 laforin 和 malin 之间相互作用的机制。该复合物的调节对于维持正常的糖原代谢是必要的。重要的是,Ser(25)在一些 LD 患者中发生突变(S25P),我们的结果开始阐明这些患者的疾病机制。