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

Novel Treatments for Lysosomal Neurological Disorders: Targeting Microglia Cells and Misfolded 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：
369322
负责人：
Pshezhetsky Alexey V
金额：
$52.11万
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：
2017
资助国家：
加拿大
项目状态：
已结题
起止时间：
2017-09-01 至 2022-09-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=369322&lang=en
关键词：
Heparan Sulfate Lysosomal Storage Disorders Lysosome Microglial Cell Missense Mutations Molecular Chaperones Mucopolysaccharidosis

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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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