Pathogenic Huntingtin Repeat Expansions in Patients with Frontotemporal Dementia and Amyotrophic Lateral Sclerosis

Maria L. Waldo, Per M. Johansson, Christer F. Nilsson, The American Genome Center (TAGC), The FALS Sequencing Consortium, The Genomics England Research Consortium, The International ALS/FTD Genomics Consortium (iAFGC), International FTD-Genomics Consortium (IFGC), The International LBD Genomics Consortium (iLBDGC), The NYGC ALS Consortium, The PROSPECT Consortium

Research output: Contribution to journalArticlepeer-review

Abstract

Using large-scale whole-genome sequencing, Dewan et al. identify pathogenic HTT repeat expansions in patients diagnosed with FTD/ALS neurodegenerative disorders. Autopsies confirm the TDP-43 pathology expected in FTD/ALS and show polyglutamine inclusions within the frontal cortices but no striatal degeneration. These data broaden the phenotype resulting from HTT repeat expansions.

Original languageEnglish
JournalNeuron
DOIs
Publication statusPublished - 2020

Subject classification (UKÄ)

  • Neurology

Free keywords

  • amyotrophic lateral sclerosis
  • frontotemporal dementia
  • huntingtin
  • repeat expansions
  • whole-genome sequencing

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