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Enzyme Replacement Therapy in Niemann-Pick Disease
University of Arizona researchers have developed an acid sphingomyelinas knockout (aSMaseKO) mouse model as a proof-of-concept for using the mutant enzyme in enzyme replacement studies for Niemann-Pick disease (NPD). NPD is a rare, inherited disease that affects the body's ability to metabolize fat within cells. The inventors suggest that a mutant...
Published: 10/26/2025
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Inventor(s):
Ashley Snider
,
Yusuf Hannun
,
Russel Jenkins
,
Benjamin Newcomb
Keywords(s):
Category(s):
Technology Classifications > Life Sciences > Therapeutics