The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Mar. 17, 2020
Filed:
Jul. 16, 2018
Children's Hospital Medical Center, Cincinnati, OH (US);
Ying Sun, Mason, OH (US);
Zhaolin Wang, Wellesley, MA (US);
Children's Hospital Medical Center, Cincinnati, OH (US);
Abstract
Disclosed herein are β-glucosidase (GCase) chaperones and methods of using GCase chaperones in an individual in need thereof. GBA1 mutations lead to GCase deficiency and substrate accumulation, causing Gaucher disease. Currently, no FDA or EMA-approved therapeutic for neuronopathic Gaucher disease is available. Improved GCase activity in brain cells using a chaperone may reduce substrate accumulation and associated pathology. Disclosed herein are novel non-inhibitory chaperone compounds of GCase that have properties of a central nervous system drug. Those compounds effectively restored mutant GCase activity by stabilizing protein and enhancing lysosomal localization and may be useful for chaperone therapy to treat neuronopathic Gaucher disease and likely to Parkinson's disease.