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Title of Journal: Cell Mol Life Sci

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Abbravation: Cellular and Molecular Life Sciences

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Publisher

SP Birkhäuser Verlag Basel

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DOI

10.1016/0002-9343(80)90037-6

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ISSN

1420-9071

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Chemical chaperone therapy for GSubscriptM1/Sub

Authors: Y Suzuki
Publish Date: 2008/01/19
Volume: 65, Issue: 3, Pages: 351-353
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Abstract

We have proposed a chemical chaperone therapy for lysosomal diseases based on a paradoxical phenomenon that an exogenous competitive inhibitor of low molecular weight stabilizes the target mutant molecule and restores its catalytic activity as a molecular chaperone intracellularly After Fabry disease experiments we investigated a new synthetic chaperone compound Noctyl4epiβvalienamine NOEV in a GM1gangliosidosis model mice Orally administered NOEV entered the brain through the bloodbrain barrier enhanced βgalactosidase activity reduced the substrate storage and clinically improved neurological deterioration We hope that chemical chaperone therapy will prove useful for some patients with GM1gangliosidosis and potentially other lysosomal storage diseases with central nervous system involvement

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