Who is at Risk of Parkinson Disease? Refining the Preclinical Phase of GBA1 and LRRK2 Variant Carriers: a Clinical, Biochemical, and Imaging Approach

Curr Neurol Neurosci Rep. 2023 Apr;23(4):121-130. doi: 10.1007/s11910-023-01259-1. Epub 2023 Mar 7.

Abstract

Purpose of review: Genetic variants in GBA1 and LRRK2 genes are the commonest genetic risk factor for Parkinson disease (PD); however, the preclinical profile of GBA1 and LRRK2 variant carriers who will develop PD is unclear. This review aims to highlight the more sensitive markers that can stratify PD risk in non-manifesting GBA1 and LRRK2 variant carriers.

Recent findings: Several case-control and a few longitudinal studies evaluated clinical, biochemical, and neuroimaging markers within cohorts of non-manifesting carriers of GBA1 and LRRK2 variants. Despite similar levels of penetrance of PD in GBA1 and LRRK2 variant carriers (10-30%), these individuals have distinct preclinical profiles. GBA1 variant carriers at higher risk of PD can present with prodromal symptoms suggestive of PD (hyposmia), display increased α-synuclein levels in peripheral blood mononuclear cells, and show dopamine transporter abnormalities. LRRK2 variant carriers at higher risk of PD might show subtle motor abnormalities, but no prodromal symptoms, higher exposure to some environmental factors (non-steroid anti-inflammatory drugs), and peripheral inflammatory profile. This information will help clinicians tailor appropriate screening tests and counseling and facilitate researchers in the development of predictive markers, disease-modifying treatments, and selection of healthy individuals who might benefit from preventive interventions.

Keywords: Carrier; GBA1; Glucocerebrosidase; LRRK2; Parkinson disease; Prodromal.

Publication types

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

MeSH terms

  • Glucosylceramidase / genetics
  • Heterozygote
  • Humans
  • Leucine-Rich Repeat Serine-Threonine Protein Kinase-2 / genetics
  • Leukocytes, Mononuclear
  • Longitudinal Studies
  • Mutation
  • Parkinson Disease* / diagnostic imaging
  • Parkinson Disease* / genetics
  • Prodromal Symptoms

Substances

  • Glucosylceramidase
  • Leucine-Rich Repeat Serine-Threonine Protein Kinase-2
  • LRRK2 protein, human
  • GBA protein, human