Substrate Deprivation Therapy to Reduce Glycosaminoglycan Synthesis Improves Aspects of Neurological and Skeletal Pathology in MPS I Mice
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MDPI AG
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http://www.mdpi.com/2079-9721/5/1/5/pdf
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5. Bone Marrow Transplantation as Effective Treatment of Central Nervous System Disease in Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Adrenoleukodystrophy, Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and Sly Syndromes, and Gaucher Disease Type III;Krivit;Curr. Opin. Neurol.,1999
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