公开(公告)号：US11883506B2

公开(公告)日：2024-01-30

申请号：US17670389

申请日：2022-02-11

申请人： Spacecraft Seven, LLC

发明人： Christopher Dean Herzog ,  Chester Bittencort Sacramento ,  Raj Prabhakar ,  David Ricks

IPC分类号： A61K48/00 ,  C12N15/864 ,  C12N15/113 ,  C12N15/02 ,  A61P9/00 ,  C07K14/005 ,  C12N15/86 ,  A61K38/00

CPC分类号： A61K48/005 ,  C07K14/005 ,  C12N15/113 ,  C12N15/86 ,  A61K38/00 ,  C12N2750/14143

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.