Treating Lysosomal Storage Diseases that Affect the Central Nervous System: Overcoming the Blood_Brain Barrier.

The article discusses the complexity of neuropathology in lysosomal storage diseases (LSD) and blood-brain barrier (BBB), with particular focus on the developments in therapeutics targeting the central nervous system (CNS) of LSDs. It discusses LSD neuropathy, which has been attributed to diseases in CNS and peripheral nervous system (PNS). It suggests that the discovery of adeno-associated viral (AAV9) trans-BBB neutropism provides promise for LSD treatment through systemic vector delivery.