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The effects of intracisternal enzyme replacement versus sham treatment on central neuropathology in preclinical canine fucosidosis
BACKGROUND: Fucosidosis results from lack of α-L-fucosidase activity, with accumulation of fucose-linked substrates in the nervous system and viscera leading to progressive motor and mental deterioration, and death. The naturally occurring dog model of fucosidosis was used to evaluate the neuropatho...
| Autores principales: | , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2015
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4632352/ https://www.ncbi.nlm.nih.gov/pubmed/26537923 http://dx.doi.org/10.1186/s13023-015-0357-z |