Thursday, February 20, 2014
Publication and contact
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,
All contents Copyright © 1993-2017 BioCentury Publications, Inc. ALL RIGHTS RESERVED. All use of this Web Site and its contents is governed by the BioCentury User Agreement
. The contents of this Web Site are protected under U.S. and foreign copyright and intellectual property laws, and no part of this Web Site or its contents may be photocopied, reproduced or retransmitted in any form without the written consent of BioCentury, which may be requested from Reprints/Permissions
. BioCentury®; The Bernstein Report on BioBusiness™; The BioCentury 100™; The Clear Route to ROI™; Because Real Intelligence is Hard to Find™; BCIQ™; and BioPharma's Knowledge Center™; are trademarks of BioCentury Publications, Inc., P.O. Box 1246, San Carlos, CA 94070. SciBX® and SciBX: Science-Business eXchange® are trademarks of Nature America, Inc. that are jointly used by BioCentury Publications, Inc. and Nature America, Inc.
Thanks for purchasing this article.
Please bookmark this url for future reference.
We have also sent you a confirming e-mail with a receipt and this same url for your
close [ X ]