Mezghrani Alexandre, Monteil Arnaud, Watschinger Katrin, Sinnegger-Brauns Martina J, Barrère Christian, Bourinet Emmanuel, Nargeot Joël, Striessnig Jörg, Lory Philippe
Centre National de la Recherche Scientifique, Unité Mixte de Recherche 5203, Institut de Génomique Fonctionnelle, Institut National de la Santé et de la Recherche Médicale, 34094 Montpellier, France.
J Neurosci. 2008 Apr 23;28(17):4501-11. doi: 10.1523/JNEUROSCI.2844-07.2008.
Channelopathies are often linked to defective protein folding and trafficking. Among them, the calcium channelopathy episodic ataxia type-2 (EA2) is an autosomal dominant disorder related to mutations in the pore-forming Ca(v)2.1 subunit of P/Q-type calcium channels. Although EA2 is linked to loss of Ca(v)2.1 channel activity, the molecular mechanism underlying dominant inheritance remains unclear. Here, we show that EA2 mutants as well as a truncated form (D(I-II)) of the Ca(v)3.2 subunit of T-type calcium channel are misfolded, retained in the endoplasmic reticulum, and subject to proteasomal degradation. Pulse-chase experiments revealed that misfolded mutants bind to nascent wild-type Ca(v) subunits and induce their subsequent degradation, thereby abolishing channel activity. We conclude that this destructive interaction mechanism promoted by Ca(v) mutants is likely to occur in EA2 and in other inherited dominant channelopathies.
离子通道病常与蛋白质折叠和运输缺陷有关。其中,钙通道病发作性共济失调2型(EA2)是一种常染色体显性疾病,与P/Q型钙通道的孔形成Ca(v)2.1亚基突变有关。尽管EA2与Ca(v)2.1通道活性丧失有关,但其显性遗传的分子机制仍不清楚。在此,我们表明,EA2突变体以及T型钙通道Ca(v)3.2亚基的截短形式(D(I-II))发生错误折叠,滞留在内质网中,并被蛋白酶体降解。脉冲追踪实验表明,错误折叠的突变体与新生的野生型Ca(v)亚基结合,并诱导其随后降解,从而消除通道活性。我们得出结论,这种由Ca(v)突变体促进的破坏性相互作用机制可能在EA2和其他遗传性显性离子通道病中发生。