Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I.
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Sci Transl Med. 2010 Dec 1;2(60):60ra89. doi: 10.1126/scitranslmed.3001380.
Sci Transl Med. 2010.
PMID: 21123810
Free PMC article.