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

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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Bibliographic Details
Main Authors: Christiane S. Hampe, Jacob Wesley, Troy C. Lund, Paul J. Orchard, Lynda E. Polgreen, Julie B. Eisengart, Linda K. McLoon, Sebahattin Cureoglu, Patricia Schachern, R. Scott McIvor
Format: Article
Language:English
Published: MDPI AG 2021-01-01
Series:Biomolecules
Subjects:
mucopolysaccharidosis type I
Hurler syndrome
enzyme replacement therapy
hematopoietic stem cell transplantations
animal models
experimental therapies
Online Access:https://www.mdpi.com/2218-273X/11/2/189

Internet

https://www.mdpi.com/2218-273X/11/2/189

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