TREM2 mutations implicated in neurodegeneration impair cell surface transport and phagocytosis

Gernot Kleinberger, Yoshinori Yamanishi, Marc Suárez-Calvet, Eva Czirr, Ebba Lohmann, Elise Cuyvers, Hanne Struyfs, Nadine Pettkus, Andrea Wenninger-Weinzierl, Fargol Mazaheri, Sabina Tahirovic, Alberto Lleó, Daniel Alcolea, Juan Fortea, Michael Willem, Sven Lammich, José L. Molinuevo, Raquel Sánchez-Valle, Anna Antonell, Alfredo RamirezMichael T. Heneka, Kristel Sleegers, Julie Van Der Zee, Jean Jacques Martin, Sebastiaan Engelborghs, Asli Demirtas-Tatlidede, Henrik Zetterberg, Christine Van Broeckhoven, Hakan Gurvit, Tony Wyss-Coray, John Hardy, Marco Colonna, Christian Haass

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573 Scopus citations

Abstract

Genetic variants in the triggering receptor expressed on myeloid cells 2 (TREM2) have been linked to Nasu-Hakola disease, Alzheimer's disease (AD), Parkinson's disease, amyotrophic lateral sclerosis, frontotemporal dementia (FTD), and FTD-like syndrome without bone involvement. TREM2 is an innate immune receptor preferentially expressed bymicroglia and is involved in inflammation and phagocytosis. Whether and how TREM2 missense mutations affect TREM2 function is unclear. We report that missensemutations associated with FTD and FTD-like syndrome reduce TREM2 maturation, abolish shedding by ADAM proteases, and impair the phagocytic activity of TREM2-expressing cells. As a consequence of reduced shedding, TREM2 is virtually absent in the cerebrospinal fluid (CSF) and plasma of a patient with FTD-like syndrome. A decrease in soluble TREM2 was also observed in the CSF of patients with AD and FTD, further suggesting that reduced TREM2 function may contribute to increased risk for two neurodegenerative disorders.

Original languageEnglish
Article number243ra86
JournalScience translational medicine
Volume6
Issue number243
DOIs
StatePublished - Jul 2 2014

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