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KMID : 1146120160020010017
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Journal of Mucopolysaccharidosis and Rare Diseases
2016 Volume.2 No. 1 p.17 ~ p.18
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Development of New Strategies for Enzyme Replacement Therapy for Lysosomal Storage Disorder
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Ko Ah-Ra
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Abstract
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Enzyme replacement therapy (ERT) is a well-established means of treating lysosomal storage disease (LSD). However, classical IV infusion based ERT method produces less than ideal results, especially, CNS defects and quality of life in patients. To improve these main problems of parental IV formulation for LSDs, we investigate modified ERT method and evaluated the efficacy in animal model.
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KEYWORD
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MPS II, Intraventricular (ICV), Subcutaneous (SC), Enzyme replacement therapy
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