Tau and TDP-43 synergy: a novel therapeutic target for sporadic late-onset Alzheimer's disease

Geroscience. 2021 Aug;43(4):1627-1634. doi: 10.1007/s11357-021-00407-0. Epub 2021 Jun 29.

Abstract

Alzheimer's disease (AD) is traditionally defined by the presence of two types of protein aggregates in the brain: amyloid plaques comprised of the protein amyloid-β (Aβ) and neurofibrillary tangles containing the protein tau. However, a large proportion (up to 57%) of AD patients also have TDP-43 aggregates present as an additional comorbid pathology. The presence of TDP-43 aggregates in AD correlates with hippocampal sclerosis, worse brain atrophy, more severe cognitive impairment, and more rapid cognitive decline. In patients with mixed Aβ, tau, and TDP-43 pathology, TDP-43 may interact with neurodegenerative processes in AD, worsening outcomes. While considerable progress has been made to characterize TDP-43 pathology in AD and late-onset dementia, there remains a critical need for mechanistic studies to understand underlying disease biology and develop therapeutic interventions. This perspectives article reviews the current understanding of these processes from autopsy cohort studies and model organism-based research, and proposes targeting neurotoxic synergies between tau and TDP-43 as a new therapeutic strategy for AD with comorbid TDP-43 pathology.

Keywords: Alzheimer’s disease; Amyloid-β (Aβ); Frontotemporal lobar degeneration (FTLD); Limbic-predominant age-related TDP-43 encephalopathy (LATE); TDP-43; Tau.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Review

MeSH terms

  • Alzheimer Disease* / drug therapy
  • Amyloid beta-Peptides
  • DNA-Binding Proteins
  • Humans
  • Neurofibrillary Tangles
  • tau Proteins

Substances

  • Amyloid beta-Peptides
  • DNA-Binding Proteins
  • TARDBP protein, human
  • tau Proteins