نتایج جستجو برای: pick type c

تعداد نتایج: 2248990  

Journal: :Journal of Neurology, Neurosurgery & Psychiatry 2003

Journal: :Journal of Inherited Metabolic Disease 2010

Journal: :Proceedings of the National Academy of Sciences of the United States of America 2003
Dennis C Ko Jonathan Binkley Arend Sidow Matthew P Scott

The neurodegenerative disease Niemann-Pick Type C2 (NPC2) results from mutations in the NPC2 (HE1) gene that cause abnormally high cholesterol accumulation in cells. We find that purified NPC2, a secreted soluble protein, binds cholesterol specifically with a much higher affinity (K(d) = 30-50 nM) than previously reported. Genetic and biochemical studies identified single amino acid changes tha...

Journal: :Journal of Veterinary Internal Medicine 1994

Journal: :Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease 2012

2016
Steven U. Walkley Cristin D. Davidson Jonathan Jacoby Philip D. Marella Elizabeth A. Ottinger Christopher P. Austin Forbes D. Porter Charles H. Vite Daniel S. Ory

Rare disease represents one of the most significant issues facing the medical community and health care providers worldwide, yet the majority of these disorders never emerge from their obscurity, drawing little attention from the medical community or the pharmaceutical industry. The challenge therefore is how best to mobilize rare disease stakeholders to enhance basic, translational and clinica...

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