Search Results - "Dierenfeld, Ashley D"
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Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I
Published in Science translational medicine (01-12-2010)“…Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease caused by loss of activity of α-l-iduronidase and attendant accumulation of the…”
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Journal Article -
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Mucopolysaccharidosis Type IIIB and GalNAc Transferase Double Knockout Mice Manifest Early Onset Fatal Leukodystrophy
Published in The FASEB journal (01-04-2010)“…Mucopolysaccharidosis type IIIB (MPS IIIB) causes progressive mental degeneration and early death due to lysosomal accumulation of heparan sulfate resulting…”
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Journal Article