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Enzyme Replacement Therapy in Niemann-Pick Disease
The inventors have developed an aSMaseKO mouse model that can be utilized as a proof-of-concept for using the mutant enzyme in enzyme replacement studies in patients with Niemann-Pick disease (NPD). NPD is a rare, inherited disease that affects the body's ability to metabolize fat (cholesterol and lipids) within cells. The inventors suggest that...
Published: 8/14/2025
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Inventor(s): Ashley Snider, Yusuf Hannun, Russel Jenkins, Benjamin Newcomb
Keywords(s):
Category(s): Technology Classifications > Life Sciences > Therapeutics
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