{-}Cellular and biochemical response to chaperone versus substrate reduction therapies in neuropathic Gaucher disease{-}
Margarita M. Ivanova, Julia Dao, Neil Kasaci, Benjamin Adewale, Shaista Nazari, Lauren Noll, Jacqueline Fikry, Armaghan Hafez Sanati, Özlem Göker-Alpan
This study compares the effects of chaperone (AMB) and substrate reduction (EGT) therapies on GCase activity, autophagy, and mitochondrial function in GD2-3 patient-derived cells.
PLoS ONE · Oct 25, 2021Peer-reviewedMolecular Biology