The cellular and extracellular proteomic signature of human dopaminergic neurons carrying the LRRK2 G2019S mutation.

ABSTRACT:

Background

Extracellular vesicles are easily accessible in various biofluids and allow the assessment of disease-related changes in the proteome. This has made them a promising target for biomarker studies, especially in the field of neurodegeneration where access to diseased tissue is very limited. Genetic variants in the LRRK2 gene have been linked to both familial and sporadic forms of Parkinson's disease. With LRRK2 inhibitors entering clinical trials, there is an unmet need for biomarkers that reflect LRRK2-specific pathology and target engagement.

Methods

In this study, we used induced pluripotent stem cells derived from a patient with Parkinson's disease carrying the LRRK2 G2019S mutation and an isogenic gene-corrected control to generate human dopaminergic neurons. W

SUBMITTER: Knab F 

PROVIDER: S-EPMC11669673 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature