[Neurodegenerative disease associated with a mutation of codon 279 (N279K) in exon 10 of Tau protein]

Bull Acad Natl Med. 2000;184(4):799-809; discussion 809-11.
[Article in French]

Abstract

Frontotemporal dementia and Parkinsonism linked to chromosome 17 (FTDP-17) are related to pathogenic mutations of the Tau gene. One of these, located at codon 279, results in an asparagine to lysine substitution. It was detected in three unrelated families from different origins. This mutation affects splicing, allowing exon 10 to be incorporated more frequently in the Tau transcripts, causing an abnormal preponderance of three-over four-repeat isoforms in soluble tau and the presence of the four-repeat isoforms in the insoluble tau. To better understand this newly described pathology, we analysed data from the three previously reported families. The American family, described as "pallido-ponto-nigral degeneration" is a large family which has been extensively studied (13 neuropathological studies). The Japanese family was initially presented as "pallidonigroluysian degeneration with iron deposition" and recently found to be related to N279 K mutation. We reported clinical, pathological and genetic data from the French family. Clinical particularities are ocular movements alterations with vertical supranuclear palsy, extrapyramidal signs (rigidity, dyskinesia, with atypical resting and postural tremor) and progressive dementia. Partial or no L-DOPA responsiveness is noted. These features led to discuss progressive supranuclear palsy, in some cases. There is no amyotrophy, nor any sensibility to neuroleptics, both signs being observed in other FTDP-17 syndromes. Neuropathology and immunohistochemistry confirm the presence of Tau immunolabeled inclusions, affecting mainly neurons in brain stem nuclei and glial cells in supratentorial white matter. Neuronal loss, which is moderate in frontal and temporal cortex, is severe in substantia nigra and globus pallidum. It is variable in other subcortical structures. In these structures, it is associated with iron deposition. This latter may participate in the degenerative process of cells and led to death in some specific neurons. The selectivity of neuronal death in hereditary diseases, when compared to data concerning sporadic neurodegenerative diseases which share similar clinical signs and neuropathological lesions, reinforces the hypothesis of an increased vulnerability of some neuronal populations which express specific sets of tau isoforms. Neurons particularly involved in these diseases express exclusively exon 10 + tau isoforms.

Publication types

  • English Abstract
  • Review

MeSH terms

  • Exons
  • Humans
  • Mutation*
  • Neurodegenerative Diseases / genetics*
  • tau Proteins / genetics*

Substances

  • tau Proteins