Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement

Mucopolysaccharidosis type I (MPS I) is a lysosomal disease, caused by a deficiency of the enzyme alpha-L-iduronidase (IDUA). IDUA catalyzes the degradation of the glycosaminoglycans dermatan and heparan sulfate (DS and HS, respectively). Lack of the enzyme leads to pathologic accumulation of undegr...

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Bibliografiske detaljer
Udgivet i:Biomolecules
Main Authors: Hampe, Christiane S., Wesley, Jacob, Lund, Troy C., Orchard, Paul J., Polgreen, Lynda E., Eisengart, Julie B., McLoon, Linda K., Cureoglu, Sebahattin, Schachern, Patricia, McIvor, R. Scott
Format: Artigo
Sprog:Inglês
Udgivet: MDPI 2021
Fag:
Review
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7911293/
https://ncbi.nlm.nih.gov/pubmed/33572941
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/biom11020189
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