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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: 10/28/2024   |   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