NeuroscienceMolecular Genetics and MetabolismPeer-reviewed
Substrate reduction therapy in a Drosophila melanogaster model of Sanfilippo syndrome
Sher Li Tan, Daniel Neumann, Paul J. Trim…·Adeline A. Lau
This study tests a novel substrate reduction therapy using a GlcNAc analogue inhibitor in Sanfilippo syndrome models, showing reduced heparan sulfate accumulation in cells and Drosophila.
DOI 10.1016/j.ymgme.2025.109112