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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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Detalhes bibliográficos
Publicado no:PLoS One
Main Authors: Tkachyova, Ilona, Fan, Xiaolian, LamHonWah, Anne-Marie, Fedyshyn, Bohdana, Tein, Ingrid, Mahuran, Don J., Schulze, Andreas
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2016
Assuntos:
Acesso em linha: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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