Adeno-associated virus with engineered capsid
AssigneeTenaya Therapeutics, Inc.
InventorChristopher A. REID
Life of this patent
Estimating the term…
Abstract
The present disclosure provides recombinant adeno-associated virus (rAAV) virions with an engineered capsid protein identified using a high-throughput sequencing screen or rational design. In particular, the disclosure provides AAV5 virions and capsid proteins with increased transduction efficiency in cardiac cells, increased cell-type selectivity, and/or other desirable properties.
Claims11
A method for treating an arrhythmogenic right ventricular cardiomyopathy (ARVC) in a subject in need thereof, comprising administering a therapeutically effective amount of a composition comprising (i) a recombinant adeno-associated virus (rAAV) virion comprising a heterologous nucleic acid encoding a plakophilin 2 (PKP2) polypeptide operatively linked to a cardiac-specific promoter;
- and (ii) a pharmaceutically acceptable carrier or excipient.
-21. (canceled)
Dependencies are inferred from the claim text.