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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7911293/
https://www.ncbi.nlm.nih.gov/pubmed/33572941
http://dx.doi.org/10.3390/biom11020189

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7911293/
https://www.ncbi.nlm.nih.gov/pubmed/33572941
http://dx.doi.org/10.3390/biom11020189

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