Clinicopathologic variability of the GRN A9D mutation, including amyotrophic lateral sclerosis

Neurology. 2013 May 7;80(19):1771-7. doi: 10.1212/WNL.0b013e3182919059. Epub 2013 Apr 17.

Abstract

Objective: We examined the clinical and pathologic phenotypes of GRN mutation carriers with the pathogenic A9D (g.26C>A) missense mutation.

Methods: Three patients with GRN A9D mutations were evaluated clinically and came to autopsy with subsequent neuropathologic examination.

Results: The clinical diagnoses of patients with GRN A9D mutations were amyotrophic lateral sclerosis, atypical extrapyramidal disorder, and behavioral variant frontotemporal dementia. Immunohistochemistry for TAR DNA-binding protein 43 (TDP-43) revealed variability in morphology and distribution of pathology. One patient had notable involvement of motor neurons in the spinal cord as well as type B TDP-43, whereas 2 other patients had type A TDP-43.

Conclusions: The clinical presentation of the GRN A9D missense mutation is not restricted to behavioral variant frontotemporal dementia and may include aphasia, extrapyramidal features, and, notably, amyotrophic lateral sclerosis.

Publication types

  • Case Reports
  • Research Support, N.I.H., Extramural

MeSH terms

  • Aged
  • Amyotrophic Lateral Sclerosis / diagnosis*
  • Amyotrophic Lateral Sclerosis / genetics*
  • Amyotrophic Lateral Sclerosis / pathology
  • Basal Ganglia Diseases / diagnosis
  • Basal Ganglia Diseases / genetics
  • Basal Ganglia Diseases / pathology
  • Brain / pathology*
  • Female
  • Frontotemporal Dementia / diagnosis
  • Frontotemporal Dementia / genetics
  • Frontotemporal Dementia / pathology
  • Genetic Variation / genetics*
  • Humans
  • Intercellular Signaling Peptides and Proteins / genetics*
  • Male
  • Middle Aged
  • Mutation, Missense / genetics*
  • Progranulins

Substances

  • GRN protein, human
  • Intercellular Signaling Peptides and Proteins
  • Progranulins