Substrate Deprivation Therapy to Reduce Glycosaminoglycan Synthesis Improves Aspects of Neurological and Skeletal Pathology in MPS I Mice

Substrate Deprivation Therapy to Reduce Glycosaminoglycan Synthesis Improves Aspects of Neurological and Skeletal Pathology in MPS I Mice

Mucopolysaccharidosis type I (MPS I) is the most common form of the MPS group of genetic diseases. MPS I results from a deficiency in the lysosomal enzyme α-l-iduronidase, leading to accumulation of undegraded heparan and dermatan sulphate glycosaminoglycan (GAG) chains in patient cells. MPS childre...

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Bibliographic Details
Main Authors: Ainslie L. K. Derrick-Roberts, Matilda R. Jackson, Carmen E. Pyragius, Sharon Byers
Format: Article
Language:English
Published: MDPI AG 2017-02-01
Series:Diseases
Subjects:
mucopolysaccharidosis type I
substrate deprivation
rhodamine B
lysosomal storage disorder
glycosaminoglycans
Online Access:http://www.mdpi.com/2079-9721/5/1/5

Internet

http://www.mdpi.com/2079-9721/5/1/5

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