Patent ID: 11883506
Assignee: SPACECRAFT SEVEN, LLC
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 0:
1. A method of treating arrhythmogenic cardiomyopathy (ACM) in a subject having a mutation in a Plakophilin-2 (PKP2) gene, comprising:
administering to the subject by intravenous administration, intracoronary administration, intracardiac administration, or cardiac catheterization, a recombinant adeno-associated virus (rAAV) vector comprising a vector genome,
wherein the vector genome comprises an expression cassette and flanking AAV inverted terminal repeats (ITRs), the expression cassette comprising a polynucleotide comprising:
a polynucleotide sequence encoding a PKP2;
a promoter sequence, wherein the promoter sequence is operatively linked to the polynucleotide sequence encoding the PKP2; and
a polyA sequence,

wherein the rAAV vector is a recombinant AAVrh74 vector,
wherein the promoter sequence comprises a human cardiac troponin T (hTNNT2) promoter and exon 1 of the hTNNT2 gene, wherein the promoter sequence shares at least 98% polynucleotide sequence identity with SEQ ID NO: 32; and
wherein the PKP2 is PKP2 isoform A and shares at least 90% polypeptide sequence identity with SEQ ID NO: 1.