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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Detalles Bibliográficos
Autores principales: Derrick-Roberts, Ainslie L. K., Jackson, Matilda R., Pyragius, Carmen E., Byers, Sharon
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2017
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5456338/
https://www.ncbi.nlm.nih.gov/pubmed/28933358
http://dx.doi.org/10.3390/diseases5010005

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5456338/
https://www.ncbi.nlm.nih.gov/pubmed/28933358
http://dx.doi.org/10.3390/diseases5010005

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