Xuefang Pan

Inhibition of macrophage neuraminidase 1 protects against immune thrombocytopenia by limiting platelet clearance

This study shows that inhibiting macrophage NEU1 reduces platelet clearance in ITP by limiting phagocytosis, suggesting NEU1 as a therapeutic target.

Extracellular vesicles derived from cells overexpressing HGSNAT rescue defects in Mucopolysaccharidosis IIIC neurons

The paper shows that extracellular vesicles from HGSNAT-overexpressing cells can rescue MPS IIIC neuron defects by delivering the enzyme to lysosomes.

Synthetic analogue of adrenocorticotropic hormone, ACTH(4-7)PGP delays neurological manifestations in diseases of mucopolysaccharidosis III spectrum by reducing neuroinflammation and rescuing neurotransmission, synaptogenesis, and axonal demyelination

The paper tests a synthetic peptide ACTH(4-7)PGP in MPS III mouse models, showing it improves synaptic function, reduces neuroinflammation, and extends lifespan.

Secondary deficiency of neuraminidase 1 contributes to CNS pathology in neurological mucopolysaccharidoses via hypersialylation of brain glycoproteins

This study shows that reduced NEU1 activity in neurological MPS leads to hypersialylation of brain glycoproteins, contributing to CNS pathology through disrupted synaptogenesis and behavior.

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.

Early defects in lysosomal storage diseases disrupt excitatory synaptic transmission

This study shows that lysosomal storage diseases cause synaptic structure and neurotransmission defects before cognitive symptoms appear in mice and humans.