Search Results - wendy+westbroek

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Mouse Model Created Using Glucocerebrosidase-Deficient Neuronal Cell Line to Study Gaucher Disease Pathophysiology and Evaluate New Therapies

This technology includes a high-yield, easy-to-culture mouse neuronal cell model with nearly complete glucocerebrosidase deficiency representative of Gaucher disease (GD) to study pathophysiology and evaluate new therapies. GD is an autosomal recessive lysosomal storage disorder caused by loss-of function mutations in the GBA1 gene, which codes for...

Published: 7/25/2024 | Inventor(s): Matthew Nguyen, Wendy Westbroek, Nahid Tayebi, Ellen Sidransky

Keywords(s): Cell, DEFICIENT, Disease, Gaucher, GLUCOCEREBROSIDASE, Line, Model, Mouse, NEURONAL, VEXXXX, VPXXXX, WIXXXX, XEXXXX

Category(s): TherapeuticArea > Neurology, Application > Research Materials, TherapeuticArea > Dental, Application > Therapeutics, TherapeuticArea > Oncology, TherapeuticArea > Endocrinology, TherapeuticArea > Ophthalmology, TherapeuticArea > Cardiology, TherapeuticArea > Infectious Disease

Glucocerebrosidase Non-inhibitory Chaperones for the Treatment of Gaucher Disease, Parkinson's Disease, and Other Proteinopathies

Gaucher disease is a rare lysosomal storage disease that is characterized by a loss of function of the glucocerebrosidase (GCase) enzyme, which results in a decreased ability to degrade its lipid substrate, glucocerebroside. The intracellular build up of this lipid causes a broad range of clinical manifestations, ranging from enlarged spleen/liver...

Published: 7/25/2024 | Inventor(s): Wei Zheng, Samarjit Patnaik, Noel Southall, Ellen Sidransky, Ehud Goldin, Wendy Westbroek, Steven Rogers, Frank Schoenen, Juan Marugan

Keywords(s): CHAPERONES, Gaucher Disease, GLUCOCEREBROSIDASE, IBXXXX, NB1BXX, NBXXXX, NON-INHIBITORY, Parkinson's Disease, proteinopathy, UAXXXX, VEXXXX, WKXXXX, YAXXXX, YBXXXX

Category(s): Collaboration Sought > Collaboration, Collaboration Sought > Licensing, Application > Therapeutics, TherapeuticArea > Neurology, TherapeuticArea > Rare / Neglected Diseases

Glucocerebrosidase Activators as a Treatment for Gaucher Disease

This technology is a collection of small molecule activators of a genetically defective version of the enzyme called glucocerebrosidase (GCase), which causes Gaucher disease. Gaucher disease is a rare disease affecting 1 in 40,000 babies born. Ashkenazi Jews of eastern European descent (about 1 in 800 live births) are at particular risk of carrying...

Published: 7/25/2024 | Inventor(s): Noel Southall, Ehud Goldin, Wei Zheng, Samarjit Patnaik, Ellen Sidransky, Omid Motabar, Wendy Westbroek, Juan Marugan

Keywords(s): ACTIVATORS, Disease, Gaucher, GLUCOCEREBROSIDASE, IA1AXX, IA1XXX, IAXXXX, IB6XXX, IBXXXX, IXXXXX, treatment

Category(s): Collaboration Sought > Licensing, TherapeuticArea > Reproductive Health, Application > Research Materials, Application > Diagnostics, Application > Therapeutics, Application > Vaccines, TherapeuticArea > Cardiology