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Mucopolysaccharidosis IIIC as a protein folding disease: using animal models to study pathophysiology and explore potential therapeutic solutions

Mucopolysaccharidosis IIIC as a protein folding disease: using animal models to study pathophysiology and explore potential therapeutic solutions
粘多糖贮积症 IIIC 作为一种蛋白质折叠疾病：利用动物模型研究病理生理学并探索潜在的治疗方案
批准号：
210690
负责人：
Pshezhetsky Alexey V
金额：
$47.39万
依托单位：
Centre hospitalier universitaire Sainte-Justine (Montral, Qubec)
依托单位国家：
加拿大
项目类别：
Operating Grants
财政年份：
2010
资助国家：
加拿大
项目状态：
已结题
起止时间：
2010-09-01 至 2015-09-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=210690&lang=en
关键词：
Animal Models Enzyme Refolding Therapy Lysosomal Storage Disease Lysosome Molecular Chaperons Mucopolysaccharidosis Mutation Analysis

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中文摘要
翻译
圣菲利波氏病属于一组影响溶酶体的儿童毁灭性遗传性疾病，溶酶体是一种负责降解和再循环大分子生物的细胞室。它会导致婴幼儿智力迅速发育迟缓
英文摘要
Sanfilippo disease belongs to a group of devastating inherited diseases of children affecting the lysosome, a cellular compartment responsible for degradation and recycling of big biological molecules. It causes rapid mental retardation in infants and chi
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