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Gene silencing of EXTL2 and EXTL3 as a substrate deprivation therapy for heparan sulphate storing mucopolysaccharidoses

Neurological pathology is characteristic of the mucopolysaccharidoses (MPSs) that store heparan sulphate (HS) glycosaminoglycan (gag) and has been proven to be refractory to systemic therapies. Substrate deprivation therapy (SDT) using general inhibitors of gag synthesis improves neurological functi...

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Bibliografiske detaljer
Main Authors: Kaidonis, Xenia, Liaw, Wan Chin, Roberts, Ainslie Derrick, Ly, Marleesa, Anson, Donald, Byers, Sharon
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2010
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2987189/
https://ncbi.nlm.nih.gov/pubmed/19690583
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ejhg.2009.143
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