Profiling extracellular vesicles in Niemann Pick disease type c

This work summarizes a doctoral research project on the role of extracellular vesicles (EVs) in the rare disease Niemann Pick Disease Type C (NPC). It established miRNA expression, surface protein profiles, size, and concentration of EVs from cerebrospinal patient (CSF) and dermal fibroblasts from NPC patients and age-matched controls. These findings reveal novel biological processes at work in a childhood disease that currently has no cure.