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GENE THERAPY FOR CILIOPATHIES
专利权人:
UCL BUSINESS LTD
发明人:
Phillip Beales,Victor Hernandez
申请号:
US16610883
公开号:
US20200069753A1
申请日:
2018.05.04
申请国别(地区):
US
年份:
2020
代理人:
摘要:
There is described a vector for treating a ciliopathy such as Bardet-Biedl syndrome, wherein the vector comprises a promoter operably linked to a ciliopathy gene, wherein the vector can provide transduction of the ciliopathy gene into multiple organs, wherein the promoter is a ubiquitous promoter which can provide expression of the ciliopathy gene in the transduced organs, and wherein the ciliopathy gene encodes a functional protein corresponding to the protein that is mutated in the ciliopathy. Also described is the use of the above vector in a method of treating a ciliopathy, the method comprising administering a therapeutically effective amount of the vector to a patient suffering from a ciliopathy.
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中国工程科技知识中心
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http://www.ckcest.cn/home/

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