Extracellular vesicles derived from cells overexpressing HGSNAT rescue defects in Mucopolysaccharidosis IIIC neurons
Travis Mooree, Mahsa Taherzadeh, Xuefang Pan, Melissa Hewitt, Caroline Sodja, Dylan Layton‐Matthews, Mehrnaz Fazeli, Anahita Bakhshizadeh, Claudie Charlebois, Marina Rukhlova, Thomas M. Durcan, Anahita Bakhshizadeh Gashti, Seyyed Mehdy Elahi, Jagdeep K. Sandhu, Anna Jezierski, Alexey V. Pshezhetsky
The paper shows that extracellular vesicles from HGSNAT-overexpressing cells can rescue MPS IIIC neuron defects by delivering the enzyme to lysosomes.
bioRxiv · Jul 23, 2026 · PreprintBiochemistry