Profound lipid dysregulation in mutant TDP-43 mice is ameliorated by the glucocerebrosidase 2 inhibitor ambroxol
Sophia J. Luikinga, Alexandre Henriques, Shyuan Thieu Ngo, Thusi Rapasinghe, Jean‐Philippe Loeffler, Michael Spedding, Bradley J. Turner
This study identifies lipid dysregulation in TDP-43 mutant mice and shows ambroxol improves motor function and preserves neurons by modulating glycosphingolipid metabolism.
bioRxiv · Aug 30, 2022 · PreprintNeuroscience