Parkinson's Disease Phenotypes in Patient Neuronal Cultures and Brain Organoids Improved by 2-Hydroxypropyl-β-Cyclodextrin Treatment.

ABSTRACT:

Background

The etiology of Parkinson's disease (PD) is only partially understood despite the fact that environmental causes, risk factors, and specific gene mutations are contributors to the disease. Biallelic mutations in the phosphatase and tensin homolog (PTEN)-induced putative kinase 1 (PINK1) gene involved in mitochondrial homeostasis, vesicle trafficking, and autophagy are sufficient to cause PD.

Objectives

We sought to evaluate the difference between controls' and PINK1 patients' derived neurons in their transition from neuroepithelial stem cells to neurons, allowing us to identify potential pathways to target with repurposed compounds.

Methods

Using two-dimensional and three-dimensional models of patients' derived neurons we recapitulated PD-related phenotypes

SUBMITTER: Jarazo J 

PROVIDER: S-EPMC9291890 | biostudies-literature | 2022 Jan

REPOSITORIES: biostudies-literature