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  • Writer's pictureDr Kerri Kinghorn

The Kinghorn lab has a new preprint

We are excited to share our new research showing that pharmacological modulation of ceramide is a potential therapeutic approach in lysosomal storage disorders, such as Niemann-Pick disease, caused by SMPD1 mutations.

Left: A healthy fly embryo showing a normal air-filled respiratory tract.

Right: Embryos lacking SMPD1 display no air filling of their respiratory tract.

This is similar to the lung disease seen in patients with Types A and B Niemann-Pick disease, caused by mutations in SMPD1.


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