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Mucopolysaccharidosis I Disease: Tools for Discovery and Design of Chaperone Therapeutics Based on Unique Enzyme Production Systems and the Elucidation of the Structure of alpha-L-Iduronidase

Mucopolysaccharidosis I Disease: Tools for Discovery and Design of Chaperone Therapeutics Based on Unique Enzyme Production Systems and the Elucidation of the Structure of alpha-L-Iduronidase
I 型粘多糖贮积症：基于独特酶生产系统和阐明 α-L-艾杜糖醛酸酶结构的伴侣治疗药物的发现和设计工具
批准号：
252123
负责人：
James Michael N
金额：
$28.24万
依托单位：
University of Alberta
依托单位国家：
加拿大
项目类别：
Operating Grants
财政年份：
2012
资助国家：
加拿大
项目状态：
已结题
起止时间：
2012-03-01 至 2015-03-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=252123&lang=en
关键词：
Enzyme Enchancement Therapy Lysosomal Storage Disease Mucopolysaccharidosis Type I Neurodegenerative Disease Structural Biology

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英文摘要
The mucopolysaccharidoses (MPS) are a group of devastating genetic diseases affecting about 1 in 25,000 individuals; in their most severe forms, they lead to death sometime during the first ten years of life. Mutations in the gene encoding the lysosomal e
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