Researchers characterize a biomarker for lysosomal storage disorders

Date: Feb-05-2014Lysosomal 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.

TITLE: Relative acidic compartment volume as a lysosomal storage disorder-associated biomarker

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