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Development of a Novel Platform to Treat Neuropathic Lysosomal Storage Diseases

Development of a Novel Platform to Treat Neuropathic Lysosomal Storage Diseases
开发治疗神经性溶酶体贮积病的新平台
批准号：
345890
负责人：
Melnyk Roman
金额：
$49.01万
依托单位：
Hospital for Sick Children (Toronto)
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：
2016-03-01 至 2020-03-01
来源：
https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=345890&lang=en
关键词：
Blood-Brain-Barrier Lysosomal Storage Diseases Monogenic Diseases Protein-Based Drugs Therapeutics Transcytosis

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中文摘要
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英文摘要
Lysosomal storage diseases (LSDs) are a group of approximately 50 disorders that result from inherited defects in proteins that reside in specialized compartments of cells throughout the body called lysosomes. LSDs are characterized by the accumulation of
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