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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 |