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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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| Main Authors: | , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society for Biochemistry and Molecular Biology
2012
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| Assuntos: | |
| Acesso em linha: | 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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