换一批A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA.
第一单位：
Telethon Institute of Genetics and Medicine (TIGEM), Naples, Italy.
作者：
医学主题词
动物(Animals);载脂蛋白B类(Apolipoproteins B);血脑屏障(Blood-Brain Barrier);脑(Brain);细胞系(Cell Line);依赖病毒(Dependovirus);疾病模型, 动物(Disease Models, Animal);基因转移技术(Gene Transfer Techniques);遗传载体(Genetic Vectors);艾杜糖醛酸硫酸酯酶(Iduronate Sulfatase);肝(Liver);膜蛋白质类(Membrane Proteins);小鼠(Mice);小鼠, 近交C57BL(Mice, Inbred C57BL);黏多糖累积病Ⅲ型(Mucopolysaccharidosis III);表型(Phenotype);蛋白质工程(Protein Engineering);蛋白质结构, 三级(Protein Structure, Tertiary);重组融合蛋白质类(Recombinant Fusion Proteins);丝氨酸内肽酶类(Serine Endopeptidases);胞吞转运(Transcytosis)
DOI
10.1002/emmm.201202083
PMID
23568409
发布时间
2025-05-29
- 浏览6
EMBO molecular medicine
675-90页
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