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Gene therapy for ciliopathies
专利权人:
UCL Business LTD
发明人:
Beales, Phillip,Hernandez, Victor
申请号:
AU2018262427
公开号:
AU2018262427A1
申请日:
2018.05.04
申请国别(地区):
AU
年份:
2019
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.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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