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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Detalles Bibliográficos
Autores principales: Tkachyova, Ilona, Fan, Xiaolian, LamHonWah, Anne-Marie, Fedyshyn, Bohdana, Tein, Ingrid, Mahuran, Don J., Schulze, Andreas
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2016
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5033324/
https://www.ncbi.nlm.nih.gov/pubmed/27657498
http://dx.doi.org/10.1371/journal.pone.0162145

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5033324/
https://www.ncbi.nlm.nih.gov/pubmed/27657498
http://dx.doi.org/10.1371/journal.pone.0162145

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