Shuxian Fan

Inhibition of cathepsin B blocks amyloidogenesis in the mouse models of neurological lysosomal diseases mucopolysaccharidosis type IIIC and sialidosis

This study shows that inhibiting cathepsin B reduces amyloid deposits in mouse models of lysosomal diseases, suggesting it as a potential therapeutic target.

Heterologous HSPC transplantation rescues neuroinflammation and ameliorates peripheral manifestations in the mouse model of lysosomal transmembrane enzyme deficiency, MPS IIIC.

This study shows that heterologous HSPC transplantation reduces neuroinflammation and improves peripheral symptoms in a mouse model of MPS IIIC, but does not correct lysosomal storage in neurons.