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Publication and contact information

Endocrine/metabolic disease

Gaucher's disease

Glucosidase-b bile acid 2 (GBA2)

Mouse studies suggest inhibiting GBA2 could help treat type 1 Gaucher's disease. In a mouse model of Gaucher's disease, Gba2 knockout resulted in decreased disease severity compared with parental controls with no Gba2 alteration. Next steps include testing GBA2 inhibitors on animal models of Gaucher's disease.

SciBX 7(16); doi:10.1038/scibx.2014.461
Published online April 24, 2014

Patent and licensing status undisclosed

Mistry, P.K. et al. Proc. Natl. Acad. Sci. USA; published online March 17, 2014;
Contact: Mone Zaidi, Icahn School of Medicine at Mount Sinai,
New York, N.Y.