Thursday, February 20, 2014
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Assays & screens
Diagnosing and monitoring
treatment responses in patients with Niemann-Pick disease type C1 (NPC1)
using fluorescence-based volumetric measurement of lysosomes
A fluorescence-based assay
in B cells could help monitor treatment responses and diagnose patients with
NPC1. The assay measured uptake of a fluorescent probe by the acidic
compartment of lysosomes to determine the volume of that compartment relative
to total cell volume. In B cells from pediatric patients with NPC1, the
method identified positive correlations between the relative compartment
volume and NPC1 disease severity. In B cells from patients with NPC1
receiving Zavesca miglustat
or bone marrow transplantation, the method identified a correlation between
decreased relative compartment volume and treatment response. Future studies
could include testing the assay in patients with other lysosomal storage
Actelion Ltd.'s and UCB Group's Zavesca, a glucosylceramide synthase (GCS) inhibitor, is marketed
to treat Gaucher's disease and Niemann-Pick disease.
Published online Feb. 20, 2014
Patent and licensing status
te Vruchte, D. et al. J.
Clin. Invest.; published online Feb. 3, 2014;
Contact: Frances M. Platt, University of Oxford, Oxford,
Contact: Mario Cortina-Borja, University College London,
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