The genetics and neuropathology of amyotrophic lateral sclerosis

Acta Neuropathol. 2012 Sep;124(3):339-52. doi: 10.1007/s00401-012-1022-4. Epub 2012 Aug 2.

Abstract

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease of motor neurons leading to death from respiratory failure within about 3 years of symptom onset. A family history of ALS is obtained in about 5 % but the distinction between familial and apparently sporadic ALS is artificial and genetic factors play a role in all types. For several years, only one gene was known to have a role in ALS pathogenesis, SOD1. In the last few years there has been a rapid advance in our genetic knowledge of the causes of ALS, and the relationship of the genetic subtypes with pathological subtypes and clinical phenotype. Mutations in the gene for TDP-43 protein, TARDBP, highlight this, with pathology mimicking closely that found in other types of ALS, and a phenotypic spectrum that includes frontotemporal dementia. Mutations in the FUS gene, closely related to TDP-43, lead to a similar clinical phenotype but distinct pathology, so that the three pathological groups represented by SOD1, TARDBP, and FUS are distinct. In this review, we explore the genetic architecture of ALS, highlight some of the genes implicated in pathogenesis, and describe their phenotypic range and overlap with other diseases.

Publication types

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

MeSH terms

  • Amyotrophic Lateral Sclerosis / genetics*
  • Amyotrophic Lateral Sclerosis / pathology*
  • DNA-Binding Proteins / genetics
  • Frontotemporal Dementia / genetics
  • Frontotemporal Dementia / pathology
  • Genetic Predisposition to Disease
  • Humans
  • Motor Neurons / pathology*
  • Superoxide Dismutase / genetics*
  • Superoxide Dismutase-1

Substances

  • DNA-Binding Proteins
  • SOD1 protein, human
  • Superoxide Dismutase
  • Superoxide Dismutase-1