Method of gene transfer for the treatment of recessive catecholaminergic polymorphic ventricular tachycardia (cpvt)
AssigneeIstituti Clinici Scientifici Maugeri SpA SB
InventorsSilvia G. PRIORI, Carlo NAPOLITANO, Marco DENEGRI
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Abstract
The present invention concerns a method for the treatment of recessive Catecholaminergic Polymorphic Ventricular Tachycardia comprising delivering a gene into a cardiac cell.
Claims11
A method of treating recessive catecholaminergic polymorphic ventricular tachycardia (CPVT) in a human patient having a deleterious mutation in the gene encoding calsequestrin 2 (CASQ2) protein, said method comprising introducing a viral vector comprising a nucleic acid encoding a wild-type CASQ2 protein into a cardiac cell in said patient, wherein administration of said viral vector to said patient results in an anti-arrhythmic effect on said patient.
Dependencies are inferred from the claim text.