Research ArticleLysosomal Storage Diseases

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

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Science Translational Medicine  01 Dec 2010:
Vol. 2, Issue 60, pp. 60ra89
DOI: 10.1126/scitranslmed.3001380

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