Register for free to
view the full innovation profile.
It may be used as an assay to screen small molecules or "drugs" that override disease-associated lipid accumulation defect.
About
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.