Pathogenic huntingtin repeat expansions in patients with frontotemporal dementia and amyotrophic lateral sclerosis
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Accepted version
Author(s)
Type
Journal Article
Abstract
We examined the role of repeat expansions in the pathogenesis of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) by analyzing whole-genome sequence data from 2,442 FTD/ALS patients, 2,599 Lewy body dementia (LBD) patients, and 3,158 neurologically healthy subjects. Pathogenic expansions (range, 40–64 CAG repeats) in the huntingtin (HTT) gene were found in three (0.12%) patients diagnosed with pure FTD/ALS syndromes but were not present in the LBD or healthy cohorts. We replicated our findings in an independent collection of 3,674 FTD/ALS patients. Postmortem evaluations of two patients revealed the classical TDP-43 pathology of FTD/ALS, as well as huntingtin-positive, ubiquitin-positive aggregates in the frontal cortex. The neostriatal atrophy that pathologically defines Huntington’s disease was absent in both cases. Our findings reveal an etiological relationship between HTT repeat expansions and FTD/ALS syndromes and indicate that genetic screening of FTD/ALS patients for HTT repeat expansions should be considered.
Date Issued
2021-02
Date Acceptance
2020-11-04
Citation
Neuron, 2021, 109 (3), pp.448-460.e4
ISSN
0896-6273
Publisher
Elsevier BV
Start Page
448
End Page
460.e4
Journal / Book Title
Neuron
Volume
109
Issue
3
Copyright Statement
© 2020 Elsevier Ltd. All rights reserved. This manuscript is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International Licence http://creativecommons.org/licenses/by-nc-nd/4.0/
Sponsor
Parkinson's UK
Identifier
https://www.sciencedirect.com/science/article/pii/S0896627320308837?via%3Dihub
Grant Number
J - 1901
Subjects
Neurology & Neurosurgery
1109 Neurosciences
1701 Psychology
1702 Cognitive Sciences
Publication Status
Published
Date Publish Online
2020-11-26