Gene therapy for treating familial hypercholesterolemia

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专利权人:

The Trustees of the University of Pennsylvania

发明人:

Wilson, James M.,Rader, Daniel J.

申请号：

AU2016366560

公开号：

AU2016366560A1

申请日:

2016.12.09

申请国别(地区):

AU

年份:

2018

代理人:

摘要：

Compositions and regimens useful in reducing one or more of: LDL-cholesterol, total cholesterol, and/or fasting triglycerides in a subject, and/or modifying fractional catabolic rate (FCR) of LDL apolipoprotein B (apoB) from baseline to a selected time point after rAAV administration are provided. The method involves administering to the human subject via a peripheral vein by infusion of a suspension of replication deficient recombinant adeno-associated virus (rAAV).

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中国工程科技知识中心

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http://www.ckcest.cn/home/