Researchers characterize a biomarker for lysosomal storage disorders
Lysosomal storage disorders (LSDs) are a common cause of neurodegenerative disease in young children.
These diseases are difficult to diagnosis in the early stages; therefore, it is difficult to develop therapeutic strategies that prevent symptom onset.
In this issue of the Journal of Clinical Investigation, Frances M. Platt and colleagues at the University of Oxford devised a method for evaluating the relative volume of acidic compartments as an indicator of LSDs.
Using the commercially available pH-indicator dye, Lysotracker, the authors were able to calculate the relative acidic compartment volume in circulating B cells. Use of this metric in samples from young LSD patients revealed that the relative acidic compartment volume correlated with clinical severity, and could be used to evaluate patient response to a particular therapy.