| 专利名称 | Familial hypercholesterolemia in gene therapy for the treatment of | ||
| 申请号 | JP2018530566 | 申请日 | |
| 公开(公告)号 | JP2019504003A | 公开(公告)日 | 2016-12-09 |
| 申请(专利权)人 | 发明人 | ウイルソン ジェームス・エム; ラダー ダニエル・ジェイ | |
| 专利来源 | 国家知识产权局 | 转化方式 | |
| 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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