Work

A modulator of wild-type glucocerebrosidase improves pathogenic phenotypes in dopaminergic neuronal models of Parkinson’s disease

Year: 2019

Type: article

Abstract: ) resulted in activation of wild-type GCase and consequent reduction of GCase lipid substrates and α-synuclein in mouse brain tissue. Our findings point to activation of wild-type GCase by small-molecule modulators as a potential therapeutic approach for treating familial and sporadic forms of PD that exhibit decreased GCase activity.

Source: Science Translational Medicine

DOI: 10.1126/scitranslmed.aau6870

Authors: Lena F. Burbulla, Sohee Jeon, Jianbin Zheng, Pingping Song, Richard B. Silverman, Dimitri Krainc

Institutions: Northwestern University, Chemistry of Life Processes Institute

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: green

Funders: Michael J. Fox Foundation for Parkinson's Research, NIH Office of the Director, National Institute of Neurological Disorders and Stroke