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Novel Treatments for Lysosomal Neurological Disorders: Targeting Microglia Cells and Missfolded Proteins for Therapy of Mucopolysaccharidosis IIIC

Novel Treatments for Lysosomal Neurological Disorders: Targeting Microglia Cells and Missfolded Proteins for Therapy of Mucopolysaccharidosis IIIC
溶酶体神经疾病的新疗法：靶向小胶质细胞和错误折叠蛋白治疗粘多糖贮积症 IIIC
批准号：
366613
负责人：
Pshezhetsky Alexey V
金额：
$7.29万
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：
2016
资助国家：
加拿大
项目状态：
已结题
起止时间：
2016-11-01 至 2017-11-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=366613&lang=en
关键词：
Heparan Sulfate Lysosomal Storage Disease Lysosome Microglial Cell Missense Mutations Molecular Chaperones Mucopolysaccharidosis

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中文摘要
翻译
Sanfilippo病属于一组破坏性的儿童遗传性疾病，影响溶酶体，溶酶体是负责降解和回收大生物分子的细胞室。它会导致婴幼儿和老年人的快速智力低下。
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 ch
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