Substrate reduction therapy in a Drosophila melanogaster model of Sanfilippo syndrome
Sher Li Tan, Daniel Neumann, Paul J. Trim, Laura J. Hewson, Nooramirah Farhana Mustaffar, Qi He, Norbert Wimmer, Marten F. Snel, Vito Ferro, Louise V. O’Keefe, Kim M. Hemsley, 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.
Molecular Genetics and Metabolism · Apr 19, 2025Peer-reviewedNeuroscience