Publications by authors named "William C Hou"
A PIKfyve modulator combined with an integrated stress response inhibitor to treat lysosomal storage diseases.
Proc Natl Acad Sci U S A
August 2024
Article Synopsis
- - Lysosomal degradation is essential for removing excess and damaged cell components, and its dysfunction is linked to various degenerative diseases, particularly lysosomal storage diseases (LSDs), where waste accumulates due to faulty lysosomal enzymes.
- - Gaucher's disease, the most prevalent LSD, occurs when glucocerebrosidase enzyme activity drops below 15%, increasing the risk for Parkinson's disease.
- - Researchers discovered that two small molecules enhancing PIKfyve activity can boost glucocerebrosidase function in Gaucher patient cells, and adding an integrated stress response inhibitor further enhances this effect, suggesting a promising treatment strategy for improving lysosomal function in LSDs.