Impact of chemical modification of sulfamidase on distribution to brain interstitial fluid and to CSF after an intravenous administration in awake, freely-moving rats

Mucopolysaccharidosis III A (MPS IIIA) is an autosomal recessive lysosomal storage disorder caused by deficiency of the enzyme sulfamidase. The disorder results in accumulation of heparan sulfate, lysosomal enlargement and cellular and organ dysfunction. Patients exhibit progressive neurodegeneratio...

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Detalles Bibliográficos
Autores principales: Janson, Juliette, Andersson, Gudrun, Bergquist, Lars, Eriksson, Maria, Folgering, Joost H.A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2019
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6939024/
https://www.ncbi.nlm.nih.gov/pubmed/31908953
http://dx.doi.org/10.1016/j.ymgmr.2019.100554

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6939024/
https://www.ncbi.nlm.nih.gov/pubmed/31908953
http://dx.doi.org/10.1016/j.ymgmr.2019.100554

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