Heat shock protein-based therapy for sphingolipidoses
Gray J., Priestman D., Wallom K-L., Petersen N., Ingemann L., Hinsby A., Arenz C., Begley D., Jäättelä M., Platt F., Kirkegaard T., Bornæs C., Drndarski S., Klein A., Jørgensen SH., Atkins J., Olsen OD., Smith DA.
Lysosomal diseases often manifest with severe systemic and central nervous system (CNS) symptoms. The existing treatment options are limited and have no or only modest efficacy against neurological manifestations of disease. We recently demonstrated that recombinant HSP70 improves the binding of several sphingolipid-degrading enzymes to their essential co-factor, bis(monoacylglycero)phosphate, in vitro