eprintid: 10043939 rev_number: 41 eprint_status: archive userid: 608 dir: disk0/10/04/39/39 datestamp: 2018-02-23 17:08:34 lastmod: 2021-09-19 23:07:04 status_changed: 2019-02-12 16:57:46 type: article metadata_visibility: show creators_name: Schneider, R creators_name: McKeever, P creators_name: Kim, T creators_name: Graff, C creators_name: van Swieten, JC creators_name: Karydas, A creators_name: Boxer, A creators_name: Rosen, H creators_name: Miller, BL creators_name: Laforce, R creators_name: Galimberti, D creators_name: Masellis, M creators_name: Borroni, B creators_name: Zhang, Z creators_name: Zinman, L creators_name: Rohrer, JD creators_name: Tartaglia, MC creators_name: Robertson, J creators_name: Genetic FTD Initiative (GENFI), . title: Downregulation of exosomal miR-204-5p and miR-632 as a biomarker for FTD: a GENFI study ispublished: pub divisions: UCL divisions: B02 divisions: C07 divisions: D07 divisions: F86 note: This is an open access article distributed in accordance with the terms of the Creative Commons Attribution (CC BY 4.0) license, which permits others to distribute, remix, adapt and build upon this work, for commercial use, provided the original work is properly cited. See: http://creativecommons.org/licenses/by/4.0/. abstract: OBJECTIVE: To determine whether exosomal microRNAs (miRNAs) in cerebrospinal fluid (CSF) of patients with frontotemporal dementia (FTD) can serve as diagnostic biomarkers, we assessed miRNA expression in the Genetic Frontotemporal Dementia Initiative (GENFI) cohort and in sporadic FTD. METHODS: GENFI participants were either carriers of a pathogenic mutation in progranulin, chromosome 9 open reading frame 72 or microtubule-associated protein tau or were at risk of carrying a mutation because a first-degree relative was a known symptomatic mutation carrier. Exosomes were isolated from CSF of 23 presymptomatic and 15 symptomatic mutation carriers and 11 healthy non-mutation carriers. Expression of 752 miRNAs was measured using quantitative PCR (qPCR) arrays and validated by qPCR using individual primers. MiRNAs found differentially expressed in symptomatic compared with presymptomatic mutation carriers were further evaluated in a cohort of 17 patients with sporadic FTD, 13 patients with sporadic Alzheimer's disease (AD) and 10 healthy controls (HCs) of similar age. RESULTS: In the GENFI cohort, miR-204-5p and miR-632 were significantly decreased in symptomatic compared with presymptomatic mutation carriers. Decrease of miR-204-5p and miR-632 revealed receiver operator characteristics with an area of 0.89 (90% CI 0.79 to 0.98) and 0.81 (90% CI 0.68 to 0.93), respectively, and when combined an area of 0.93 (90% CI 0.87 to 0.99). In sporadic FTD, only miR-632 was significantly decreased compared with AD and HCs. Decrease of miR-632 revealed an area of 0.90 (90% CI 0.81 to 0.98). CONCLUSIONS: Exosomal miR-204-5p and miR-632 have potential as diagnostic biomarkers for genetic FTD and miR-632 also for sporadic FTD. date: 2018-08 date_type: published official_url: http://doi.org/10.1136/jnnp-2017-317492 oa_status: green full_text_type: pub language: eng primo: open primo_central: open_green article_type_text: Article verified: verified_manual elements_id: 1535526 doi: 10.1136/jnnp-2017-317492 pii: jnnp-2017-317492 lyricists_name: Bocchetta, Martina lyricists_name: Rohrer, Jonathan lyricists_id: MBOCC01 lyricists_id: JDROH34 actors_name: Laslett, David actors_id: DLASL34 actors_role: owner full_text_status: public publication: Journal of Neurology, Neurosurgery & Psychiatry volume: 89 number: 8 pagerange: 851-858 issn: 1468-330X citation: Schneider, R; McKeever, P; Kim, T; Graff, C; van Swieten, JC; Karydas, A; Boxer, A; ... Genetic FTD Initiative (GENFI), .; + view all <#> Schneider, R; McKeever, P; Kim, T; Graff, C; van Swieten, JC; Karydas, A; Boxer, A; Rosen, H; Miller, BL; Laforce, R; Galimberti, D; Masellis, M; Borroni, B; Zhang, Z; Zinman, L; Rohrer, JD; Tartaglia, MC; Robertson, J; Genetic FTD Initiative (GENFI), .; - view fewer <#> (2018) Downregulation of exosomal miR-204-5p and miR-632 as a biomarker for FTD: a GENFI study. Journal of Neurology, Neurosurgery & Psychiatry , 89 (8) pp. 851-858. 10.1136/jnnp-2017-317492 <https://doi.org/10.1136/jnnp-2017-317492>. Green open access document_url: https://discovery.ucl.ac.uk/id/eprint/10043939/1/851.full.pdf