PUBLICATION

A152T tau allele causes neurodegeneration that can be ameliorated in a zebrafish model by autophagy induction

Authors
Lopez, A., Lee, S.E., Wojta, K., Ramos, E.M., Klein, E., Chen, J., Boxer, A.L., Gorno-Tempini, M.L., Geschwind, D.H., Schlotawa, L., Ogryzko, N.V., Bigio, E.H., Rogalski, E., Weintraub, S., Mesulam, M.M., Fleming, A., Coppola, G., Miller, B.L., Rubinsztein, D.C.
ID
ZDB-PUB-170324-3
Date
2017
Source
Brain : a journal of neurology   140(4): 1128-1146 (Journal)
Registered Authors
Fleming, Angeleen, Ogryzko, Nikolay
Keywords
autophagy, neurodegeneration, proteasome, tauopathy
MeSH Terms
  • Alleles
  • Animals
  • Autophagy*
  • Autophagy-Related Protein 5
  • Behavior, Animal
  • Disease Models, Animal
  • Embryo, Nonmammalian
  • Frontotemporal Dementia/genetics
  • Heredodegenerative Disorders, Nervous System/genetics*
  • Heredodegenerative Disorders, Nervous System/therapy*
  • Humans
  • Kinetics
  • Polymorphism, Single Nucleotide
  • Proteasome Endopeptidase Complex/genetics
  • RNA/biosynthesis
  • RNA/genetics
  • Supranuclear Palsy, Progressive/genetics*
  • Supranuclear Palsy, Progressive/therapy*
  • Tauopathies/genetics*
  • Tauopathies/psychology
  • Tauopathies/therapy*
  • Zebrafish*
  • Zebrafish Proteins
  • tau Proteins/genetics*
  • tau Proteins/metabolism
PubMed
28334843 Full text @ Brain
Abstract
Mutations in the gene encoding tau (MAPT) cause frontotemporal dementia spectrum disorders. A rare tau variant p.A152T was reported as a risk factor for frontotemporal dementia spectrum and Alzheimer's disease in an initial case-control study. Such findings need replication in an independent cohort. We analysed an independent multinational cohort comprising 3100 patients with neurodegenerative disease and 4351 healthy control subjects and found p.A152T associated with significantly higher risk for clinically defined frontotemporal dementia and progressive supranuclear palsy syndrome. To assess the functional and biochemical consequences of this variant, we generated transgenic zebrafish models expressing wild-type or A152T-tau, where A152T caused neurodegeneration and proteasome compromise. Impaired proteasome activity may also enhance accumulation of other proteins associated with this variant. We increased A152T clearance kinetics by both pharmacological and genetic upregulation of autophagy and ameliorated the disease pathology observed in A152T-tau fish. Thus, autophagy-upregulating therapies may be a strategy for the treatment for tauopathies.
Errata / Notes
This article is corrected by ZDB-PUB-220906-65.
Genes / Markers
Figures
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Expression
Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping