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Gaucher病的AAV基因疗法取得新进展

2020-02-13 Allan MedSci原创

Gaucher病AAV基因疗法β-葡萄糖脑苷脂酶
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Freeline是一家致力于基因疗法的生物技术公司，近日宣布将在佛罗里达州奥兰多举行的第16届WORLDSymposium™上宣布Gaucher病基因治疗计划的最新数据。

Freeline是一家致力于基因疗法的生物技术公司，近日宣布将在佛罗里达州奥兰多举行的第16届WORLDSymposium™上宣布Gaucher病基因治疗计划的最新数据。FLT200是一种针对肝脏的AAV基因疗法，用于治疗1型Gaucher病，旨在提高β-葡萄糖脑苷脂酶（GCase）的水平。可以预期，通过FLT200传递到肝脏的正常GCase基因将能够产生功能齐全的GCase酶，并防止脂肪分子的积累。临床前数据表明，在单次注射基因药物后，可以在血流中长期观察到稳定水平的全活性GCase。FLT200的生物利用度也比每两周输注一次的维拉苷酶α有效。

Gaucher病是一种溶酶体贮积病，为常染色体隐性遗传病。该病由于葡萄糖脑苷脂酶基因突变导致机体葡萄糖脑苷脂酶活性缺乏，造成其底物葡萄糖脑苷脂在肝、脾、骨骼、肺，甚至脑的巨噬细胞溶酶体中贮积，导致受累组织器官出现病变。


原始出处：

https://www.firstwordpharma.com/node/1700316?tsid=28®ion_id=5

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    2021-01-18 cooco

    #Gaucher病#

    0

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    2020-05-23 aliceclz

    #AUC#

    0

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    2020-03-17 小几洁

    #AAV基因疗法#

    0

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    2020-02-15 xuqianhua

    #AAV#

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