|
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
|
Inventor(s): Ashley Snider, Yusuf Hannun, Russel Jenkins, Benjamin Newcomb
Keywords(s):
Category(s): Technology Classifications > Life Sciences > Therapeutics
|
