TDP-43 causes neurotoxicity and cytoskeletal dysfunction in primary cortical neurons

PLoS One. 2018 May 22;13(5):e0196528. doi: 10.1371/journal.pone.0196528. eCollection 2018.

Abstract

TDP-43-mediated proteinopathy is a key factor in the pathology of amyotrophic lateral sclerosis (ALS). A potential underlying mechanism is dysregulation of the cytoskeleton. Here we investigate the effects of expressing TDP-43 wild-type and M337V and Q331K mutant isoforms on cytoskeletal integrity and function, using rat cortical neurons in vitro. We find that TDP-43 protein becomes mislocalised in axons over 24-72 hours in culture, with protein aggregation occurring at later timepoints (144 hours). Quantitation of cell viability showed toxicity of both wild-type and mutant constructs which increased over time, especially of the Q331K mutant isoform. Analysis of the effects of TDP-43 on axonal integrity showed that TDP-43-transfected neurons had shorter axons than control cells, and that growth cone sizes were smaller. Axonal transport dynamics were also impaired by transfection with TDP-43 constructs. Taken together these data show that TDP-43 mislocalisation into axons precedes cell death in cortical neurons, and that cytoskeletal structure and function is impaired by expression of either TDP-43 wild-type or mutant constructs in vitro. These data suggest that dysregulation of cytoskeletal and neuronal integrity is an important mechanism for TDP-43-mediated proteinopathy.

Publication types

  • Research Support, Non-U.S. Gov't
  • Retracted Publication

MeSH terms

  • Amyotrophic Lateral Sclerosis / etiology
  • Amyotrophic Lateral Sclerosis / genetics
  • Amyotrophic Lateral Sclerosis / metabolism
  • Animals
  • Axonal Transport / drug effects
  • Axonal Transport / physiology
  • Axons / metabolism
  • Axons / pathology
  • Cell Death
  • Cells, Cultured
  • DNA-Binding Proteins / genetics
  • DNA-Binding Proteins / metabolism*
  • DNA-Binding Proteins / toxicity
  • Growth Cones / metabolism
  • Growth Cones / pathology
  • Humans
  • Motor Neurons / drug effects
  • Motor Neurons / metabolism*
  • Motor Neurons / pathology
  • Mutant Proteins / genetics
  • Mutant Proteins / metabolism
  • Mutant Proteins / toxicity
  • Neurotoxins / genetics
  • Neurotoxins / metabolism
  • Neurotoxins / toxicity
  • Protein Aggregation, Pathological / etiology
  • Protein Aggregation, Pathological / genetics
  • Protein Aggregation, Pathological / metabolism
  • Rats
  • Recombinant Fusion Proteins / genetics
  • Recombinant Fusion Proteins / metabolism
  • Recombinant Fusion Proteins / toxicity
  • TDP-43 Proteinopathies / etiology*
  • TDP-43 Proteinopathies / genetics
  • TDP-43 Proteinopathies / metabolism
  • Transfection

Substances

  • DNA-Binding Proteins
  • Mutant Proteins
  • Neurotoxins
  • Recombinant Fusion Proteins
  • TARDBP protein, human