Restoration of Surface GM1 location Screen - a marker for rescue of lipid accumulation defect NP-C cells

Description:

Restoration of cell surface glycosphingolipid GM1 as detected by binding to cholera toxin may be used as an assay to screen for drugs capable of overriding the lipid accumulation defect

Niemann Pick Type C is a fatal neurodegenerative disorder. Intracellular accumulation of glycosphingolipids and cholesterol is detected systematically and a hallmark of disease. Reduction of intracellular sphingolipid and cholesterol stores and reversion of disease phenotype can be achieved by promoting lipid efflux from cells. The invention is the idea that restoration of cell surface glycosphingolipid GM1 as detected by binding to cholera toxin may be used as an assay to screen small molecules or "drugs" that override disease-associated lipid accumulation defect.

 
Patent Information:
Category(s):
Medical Diagnostics
For Information, Contact:
Scott Hartford
Licensing Associate
University of Notre Dame
(574) 631-7499
hartford.4@nd.edu
Inventors:
Kasturi Haldar
Crislyn D'Souza-Schorey
Keywords:
Theraputic
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