Replacing the Enzyme α-L-Iduronidase at Birth Ameliorates Symptoms in the Brain and Periphery of Dogs with Mucopolysaccharidosis Type I

Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease caused by loss of activity of α-L-iduronidase and attendant accumulation of the glycosaminoglycans dermatan and heparan sulfates. Current treatments are suboptimal and leave residual disease including corneal clouding, skeletal defo...

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Detalhes bibliográficos
Main Authors: Dierenfeld, A.D., McEntee, M.F., Vogler, C.A., Vite, C.H., Chen, A. H., Passage, M., Le, S., Shah, S., Jens, J.K., Snella, E.M., Kline, K.L., Parkes, J.D., Ware, W.A., Moran, L.E., Fales-Williams, A. J., Wengert, J.A., Whitley, R.D., Betts, D.M., Boal, A.M., Riedesel, E.A., Gross, W., Ellinwood, N.M., Dickson, P.I.
Formato: Artigo
Idioma:Inglês
Publicado em: 2010
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3075726/
https://ncbi.nlm.nih.gov/pubmed/21123810
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.3001380
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