Partial deletion of the MAPT gene: a novel mechanism of FTDP-17

Hum Mutat. 2009 Apr;30(4):E591-602. doi: 10.1002/humu.20979.

Abstract

A heterozygous genomic deletion removing exons 6 to 9 of the microtubule associated protein tau (MAPT) gene, predicting to result into a truncated protein lacking the first microtubule binding domain, was detected in a patient with frontotemporal dementia (FTD). Cell culture experiments showed that the truncated tau isoforms had a dramatic decrease in the normal binding to microtubules but acquired the ability to bind microtubule associated protein-1B (MAP-1B). This indicates that this tauopathy likely results both from a loss of function mechanism and from a deleterious gain of function by which cytoplasmic deleted forms of tau sequester another MAP. Both mechanisms could contribute to impair microtubule dynamics.

Publication types

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

MeSH terms

  • Adult
  • Alternative Splicing
  • Blotting, Western
  • Cell Line, Tumor
  • DNA Mutational Analysis
  • Fatal Outcome
  • Gene Deletion*
  • Humans
  • Immunoprecipitation
  • Male
  • Microscopy, Confocal
  • Microtubule-Associated Proteins / immunology
  • Microtubule-Associated Proteins / metabolism
  • Microtubules / metabolism
  • Protein Binding
  • Protein Isoforms / genetics
  • Protein Isoforms / metabolism
  • Tauopathies / genetics*
  • Tauopathies / metabolism
  • Tauopathies / pathology
  • Transfection
  • tau Proteins / genetics*
  • tau Proteins / metabolism

Substances

  • MAPT protein, human
  • Microtubule-Associated Proteins
  • Protein Isoforms
  • tau Proteins