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The N-terminal domain of the prion protein is required and sufficient for liquid–liquid phase separation: A crucial role of the Aβ-binding domain

Janine Kamps, Yu-Hsuan Lin, Rosario Oliva, Verian Bader, Roland Winter, Konstanze F. Winklhofer, Jörg Tatzelt

2021Journal of Biological Chemistry36 citationsDOIOpen Access PDF

Abstract

is proteolytically processed in vivo into an entirely unstructured fragment, designated N1, and the corresponding C-terminal fragment C1 harboring the globular domain. Notably, N1 contains a polybasic motif that serves as a binding site for neurotoxic Aβ oligomers. PrP can undergo LLPS; however, nothing is known how phase separation of PrP is triggered on a molecular scale. Here, we show that the intrinsically disordered N1 domain is necessary and sufficient for LLPS of PrP. Similar to full-length PrP, the N1 fragment formed highly dynamic liquid-like droplets. Remarkably, a slightly shorter unstructured fragment, designated N2, which lacks the Aβ-binding domain and is generated under stress conditions, failed to form liquid-like droplets and instead formed amorphous assemblies of irregular structures. Through a mutational analysis, we identified three positively charged lysines in the postoctarepeat region as essential drivers of condensate formation, presumably largely via cation-π interactions. These findings provide insights into the molecular basis of LLPS of the mammalian prion protein and reveal a crucial role of the Aβ-binding domain in this process.

Topics & Concepts

BiophysicsIntrinsically disordered proteinsChemistryStress granuleNeurodegenerationBinding domainPrion proteinCell biologyBiochemistryCrystallographyBiologyBinding siteMessenger RNAGeneDiseasePathologyTranslation (biology)MedicinePrion Diseases and Protein MisfoldingRNA Research and SplicingRNA regulation and disease