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...

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Udgivet i:Orphanet J Rare Dis
Main Authors: Kondagari, Gauthami Sudhamayee, Fletcher, Jessica Louise, Cruz, Rachel, Williamson, Peter, Hopwood, John J., Taylor, Rosanne Maree
Format: Artigo
Sprog:Inglês
Udgivet: BioMed Central 2015
Fag:
Research
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4632352/
https://ncbi.nlm.nih.gov/pubmed/26537923
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-015-0357-z
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