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

SCIENCE TRANSLATIONAL MEDICINE(2010)

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摘要
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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mucopolysaccharidosis type,enzyme
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