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AssigneeSpacecraft Seven, LLC
InventorsChristopher Dean HERZOG, Chester Bittencort SACRAMENTO, Raj PRABHAKAR, David RICKS
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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 AAVrh. 74 or comprise an AAV9 or AAVrh74 capsid protein 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.
1-90. (canceled)
A polynucleotide, comprising an expression cassette and flanking adeno-associated virus (AAV) inverted terminal repeats (ITRs), wherein the polynucleotide comprises a polynucleotide sequence encoding a Plakophilin-2 (PKP2), or a functional variant thereof, operatively linked to a promoter, wherein the polynucleotide shares at least 75% identity with any one of SEQ ID NOs:
Dependencies are inferred from the claim text.