Rare TBK1 variants in patients with frontotemporal dementia and amyotrophic lateral sclerosis in a Chinese cohort

Bin Jiao1,2, Qiying Sun1,3, Zhenhua Yuan2, Junling Wang1,2, Lin Zhou3,1, Xin-xiang Yan1,2, Beisha Tang4,5,6,7,2, Lu Shen8,1,2
1National Clinical Research Center for Geriatric Disorders, Central South University, Changsha, China
2Department of Neurology, Xiangya Hospital, Central South University, Changsha, China
3Department of Geriatrics Neurology, Xiangya Hospital, Central South University, Changsha, China
4Collaborative Innovation Center for Brain Science, Shanghai, China
5Collaborative Innovation Center for Genetics and Development, Shanghai, China
6Parkinson's Disease Center of Beijing Institute for Brain Disorders, Beijing, China
7Key Laboratory of Hunan Province in Neurodegenerative Disorders, Central South University, Changsha, China
8Key Laboratory of Organ Injury, Aging and Regenerative Medicine of Hunan Province, Changsha, China

Tóm tắt

Từ khóa


Tài liệu tham khảo

Kiernan MC, Vucic S, Cheah BC, Turner MR, Eisen A, Hardiman O, et al. Amyotrophic lateral sclerosis. Lancet. 2011;377(9769):942–55.

Neary D, Snowden J, Mann D. Frontotemporal dementia. Lancet Neurol. 2005;4(11):771–80.

Burrell JR, Halliday GM, Kril JJ, Ittner LM, Gotz J, Kiernan MC, et al. The frontotemporal dementia-motor neuron disease continuum. Lancet. 2016;388(10047):919–31.

Weishaupt JH, Hyman T, Dikic I. Common molecular pathways in amyotrophic lateral sclerosis and frontotemporal dementia. Trends Mol Med. 2016;22(9):769–83.

Hsieh S, Caga J, Leslie FV, Shibata M, Daveson N, Foxe D, et al. Cognitive and behavioral symptoms in ALSFTD: detection, differentiation, and progression. J Geriatr Psychiatry Neurol. 2016;29(1):3–10.

Piguet O, Hornberger M, Mioshi E, Hodges JR. Behavioural-variant frontotemporal dementia: diagnosis, clinical staging, and management. Lancet Neurol. 2011;10(2):162–72.

Neumann M, Sampathu DM, Kwong LK, Truax AC, Micsenyi MC, Chou TT, et al. Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis. Science. 2006;314(5796):130–3.

Kabashi E, Valdmanis PN, Dion P, Spiegelman D, McConkey BJ, Vande Velde C, et al. TARDBP mutations in individuals with sporadic and familial amyotrophic lateral sclerosis. Nat Genet. 2008;40(5):572–4.

DeJesus-Hernandez M, Mackenzie IR, Boeve BF, Boxer AL, Baker M, Rutherford NJ, et al. Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS. Neuron. 2011;72(2):245–56.

Johnson JO, Mandrioli J, Benatar M, Abramzon Y, Van Deerlin VM, Trojanowski JQ, et al. Exome sequencing reveals VCP mutations as a cause of familial ALS. Neuron. 2010;68(5):857–64.

Freischmidt A, Wieland T, Richter B, Ruf W, Schaeffer V, Muller K, et al. Haploinsufficiency of TBK1 causes familial ALS and fronto-temporal dementia. Nat Neurosci. 2015;18(5):631–6.

Freischmidt A, Muller K, Ludolph AC, Weishaupt JH, Andersen PM. Association of Mutations in TBK1 with sporadic and familial amyotrophic lateral sclerosis and frontotemporal dementia. JAMA Neurol. 2017;74(1):110–3.

Li F, Xu D, Wang Y, Zhou Z, Liu J, Hu S, et al. Structural insights into the ubiquitin recognition by OPTN (optineurin) and its regulation by TBK1-mediated phosphorylation. Autophagy. 2018;14(1):66–79.

Pottier C, Bieniek KF, Finch N, van de Vorst M, Baker M, Perkersen R, et al. Whole-genome sequencing reveals important role for TBK1 and OPTN mutations in frontotemporal lobar degeneration without motor neuron disease. Acta Neuropathol. 2015;130(1):77–92.

Brooks BR, El Escorial World Federation of Neurology criteria for the diagnosis of amyotrophic lateral sclerosis. Subcommittee on Motor Neuron Diseases/Amyotrophic Lateral Sclerosis of the World Federation of Neurology Research Group on Neuromuscular Diseases and the El Escorial “Clinical limits of amyotrophic lateral sclerosis” workshop contributors. J Neurol Sci. 1994;124(Suppl):96–107.

The Lund and Manchester Groups. Clinical and neuropathological criteria for frontotemporal dementia. J Neurol Neurosurg Psychiatry. 1994;57(4):416–8.

Shu S, Li XL, Liu Q, Liu F, Cui B, Liu MS, et al. Screening of the TBK1 gene in familial and sporadic amyotrophic lateral sclerosis patients of Chinese origin. Amyotroph Lateral Scler Frontotemporal Degener. 2016;17(7–8):605–7.

Tsai PC, Liu YC, Lin KP, Liu YT, Liao YC, Hsiao CT, et al. Mutational analysis of TBK1 in Taiwanese patients with amyotrophic lateral sclerosis. Neurobiol Aging. 2016;40:191.e11–6.

Gijselinck I, Van Mossevelde S, van der Zee J, Sieben A, Philtjens S, Heeman B, et al. Loss of TBK1 is a frequent cause of frontotemporal dementia in a Belgian cohort. Neurology. 2015;85(24):2116–25.

Pozzi L, Valenza F, Mosca L, Dal Mas A, Domi T, Romano A, et al. TBK1 mutations in Italian patients with amyotrophic lateral sclerosis: genetic and functional characterisation. J Neurol Neurosurg Psychiatry. 2017;88(10):869–75.

Gleason CE, Ordureau A, Gourlay R, Arthur JS, Cohen P. Polyubiquitin binding to optineurin is required for optimal activation of TANK-binding kinase 1 and production of interferon beta. J Biol Chem. 2011;286(41):35663–74.

Van Mossevelde S, Engelborghs S, van der Zee J, Van Broeckhoven C. Genotype-phenotype links in frontotemporal lobar degeneration. Nat Rev Neurol. 2018;14(6):363–78.

van der Zee J, Gijselinck I, Van Mossevelde S, Perrone F, Dillen L, Heeman B, et al. TBK1 mutation Spectrum in an extended European patient cohort with frontotemporal dementia and amyotrophic lateral sclerosis. Hum Mutat. 2017;38(3):297–309.

Van Mossevelde S, van der Zee J, Gijselinck I, Engelborghs S, Sieben A, Van Langenhove T, et al. Clinical features of TBK1 carriers compared with C9orf72, GRN and non-mutation carriers in a Belgian cohort. Brain. 2016;139(Pt 2):452–67.

Caroppo P, Camuzat A, De Septenville A, Couratier P, Lacomblez L, Auriacombe S, et al. Semantic and nonfluent aphasic variants, secondarily associated with amyotrophic lateral sclerosis, are predominant frontotemporal lobar degeneration phenotypes in TBK1 carriers. Alzheimers Dement (Amst). 2015;1(4):481–6.