A new family with frontotemporal dementia with intronic 10+3 splice site mutation in the tau gene: neuropathology and molecular effects

Neuropathol Appl Neurobiol. 2005 Aug;31(4):362-73. doi: 10.1111/j.1365-2990.2005.00629.x.

Abstract

Mutations in the tau gene cause familial frontotemporal dementia with parkinsonism linked to chromosome 17 characterized by filamentous tau protein deposits. Here we describe the clinical and neuropathological features of a case from a newly identified family with an intron 10+3-splice site mutation in the tau gene. The proband presented with severe personality changes and stereotyped speech followed by parkinsonian symptoms. He died at age 56 after a disease duration of approximately 6 years. At autopsy, there was marked frontotemporal degeneration with abundant tau-immunoreactive neuronal and glial inclusions widespread in the cortex and brainstem. RT-PCR analysis revealed a 3.7-fold increase of tau transcripts with exon 10, resulting in an 1.7-fold higher expression level of 4-repeat tau isoforms in soluble tau fractions when compared to control brains and exclusively 4-repeat tau isoforms in the sarcosyl-insoluble tau fractions. In accordance with the hypothesis that the overexpression leads to saturation of microtubule binding sites and an increase of unbound 4-repeat tau isoforms which assemble into filaments, the neuronal and glial inclusions in this case were exclusively composed of 4-repeat tau isoforms. The clinical and neuropathological data of this family are compared with results from the two other published families with the intron 10 + 3 mutation, the MSTD and the SOT 254 family.

Publication types

  • Case Reports
  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Base Sequence
  • Brain / pathology*
  • DNA Mutational Analysis
  • Dementia / genetics*
  • Dementia / pathology*
  • Female
  • Humans
  • Immunoblotting
  • Immunohistochemistry
  • Male
  • Middle Aged
  • Mutation
  • Pedigree
  • Protein Isoforms / genetics
  • RNA Splice Sites
  • Reverse Transcriptase Polymerase Chain Reaction
  • tau Proteins / genetics*

Substances

  • Protein Isoforms
  • RNA Splice Sites
  • tau Proteins