NDST1 Preferred Promoter Confirmation and Identification of Corresponding Transcriptional Inhibitors as Substrate Reduction Agents for Multiple Mucopolysaccharidosis Disorders

The stepwise degradation of glycosaminoglycans (GAGs) is accomplished by twelve lysosomal enzymes. Deficiency in any of these enzymes will result in the accumulation of the intermediate substrates on the pathway to the complete turnover of GAGs. The accumulation of these undegraded substrates in alm...

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Vydáno v:PLoS One
Hlavní autoři: Tkachyova, Ilona, Fan, Xiaolian, LamHonWah, Anne-Marie, Fedyshyn, Bohdana, Tein, Ingrid, Mahuran, Don J., Schulze, Andreas
Médium: Artigo
Jazyk:Inglês
Vydáno: Public Library of Science 2016
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Research Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5033324/
https://ncbi.nlm.nih.gov/pubmed/27657498
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0162145
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