- 专利标题： PLAKOPHILIN-2 (PKP2) GENE THERAPY USING AAV VECTOR
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申请号： US17670390申请日： 2022-02-11
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公开(公告)号： US20220168447A1公开(公告)日： 2022-06-02
- 发明人: Christopher Dean HERZOG , Chester Bittencort SACRAMENTO , Raj PRABHAKAR , David RICKS
- 申请人： Spacecraft Seven, LLC
- 申请人地址： US NJ Cranbury
- 专利权人： Spacecraft Seven, LLC
- 当前专利权人： Spacecraft Seven, LLC
- 当前专利权人地址： US NJ Cranbury
- 主分类号： A61K48/00
- IPC分类号： A61K48/00 ; C12N15/113 ; C12N15/86
摘要：
Provided herein is a gene therapy for PKP2 (Plakophilin-2), e.g. using an adeno-associated virus (AAV) vector. The promoter of the vector may be a MHCK7 promoter or a cardiac troponin T (HTNNT2) promoter. The capsid may be an AAV9 or AAVrh74 capsid or a functional variant thereof. Other promoters or capsids may be used. Further provided are methods of treatment, such as by intravenous, intracoronary, intracarotid or intracardiac administration of the rAAV vector, and other compositions and methods.
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