A Genetic Model of Substrate Reduction Therapy for Mucopolysaccharidosis

Inherited defects in the ability to catabolize glycosaminoglycans result in lysosomal storage disorders known as mucopolysaccharidoses (MPS), causing severe pathology, particularly in the brain. Enzyme replacement therapy has been used to treat mucopolysaccharidoses; however, neuropathology has rema...

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Bibliografiske detaljer
Main Authors: Lamanna, William C., Lawrence, Roger, Sarrazin, Stéphane, Lameda-Diaz, Carlos, Gordts, Philip L. S. M., Moremen, Kelley W., Esko, Jeffrey D.
Format: Artigo
Sprog:Inglês
Udgivet: American Society for Biochemistry and Molecular Biology 2012
Fag:
Glycobiology and Extracellular Matrices
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3476295/
https://ncbi.nlm.nih.gov/pubmed/22952226
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1074/jbc.M112.403360
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