A cluster of progranulin C157KfsX97 mutations in Southern Italy: clinical characterization and genetic correlations

Neurobiol Aging. 2017 Jan:49:219.e5-219.e13. doi: 10.1016/j.neurobiolaging.2016.10.008. Epub 2016 Oct 11.

Abstract

Frontotemporal lobar degeneration (FTLD) is a group of neurodegenerative diseases displaying high clinical, pathologic, and genetic heterogeneity. Several autosomal dominant progranulin (GRN) mutations have been reported, accounting for 5%-10% of FTLD cases worldwide. In this study, we described the clinical characteristics of 7 Italian patients, 5 with a diagnosis of frontotemporal dementia behavioral variant and 2 of corticobasal syndrome (CBS), carrying the GRN deletion g.101349_101355delCTGCTGT, resulting in the C157KfsX97 null mutation, and hypothesized the existence of a founder effect by means of haplotype sharing analysis. We performed plasma progranulin dosage, GRN gene sequencing, and haplotype sharing study, analyzing 10 short tandem repeat markers, spanning a region of 11.08 Mb flanking GRN on chromosome 17q21. We observed shared alleles among 6 patients for 8 consecutive short tandem repeat markers spanning a 7.29 Mb region. Therefore, also with this particular mutation, the elevated clinical variability described among GRN-mutated FTLD cases is confirmed. Moreover, this is the first study reporting the likely existence of a founder effect for C157KfsX97 mutation in Southern Italy.

Keywords: Corticobasal syndrome; Founder effect; Frontotemporal lobar degeneration; GRN; Mutation; Progranulin protein.

Publication types

  • Case Reports

MeSH terms

  • Aged
  • Alleles
  • Female
  • Founder Effect*
  • Frontotemporal Lobar Degeneration / genetics*
  • Gene Deletion
  • Gene Dosage
  • Genes, Dominant / genetics
  • Genetic Association Studies*
  • Haplotypes
  • Humans
  • Intercellular Signaling Peptides and Proteins / blood
  • Intercellular Signaling Peptides and Proteins / genetics*
  • Italy
  • Male
  • Middle Aged
  • Multigene Family / genetics*
  • Mutation / genetics*
  • Progranulins
  • Sequence Analysis, DNA
  • Tandem Repeat Sequences

Substances

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