Mybpc3 polypeptides and uses thereof
AssigneeChildren's Medical Center Corporation
InventorsWilliam T. Pu, Fujian Lu, Vassilios Bezzerides
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Abstract
Provided herein are compositions and methods for treating a disorder associated with abnormal RYR2 function (e.g., arrhythmia or heart failure). In some embodiments, method comprises administering to a subject in need thereof an effective amount of a polypeptide comprising a C-terminal domain of Cardiac Myosin binding protein C (MYBPC3) or a nucleic acid or an rAAV encoding such polypeptide.
Claims26
A method of treating a disorder associated with abnormal ryanodine receptor type 2 (RYR2) function, the method comprising administering to a subject in need thereof an effective amount of a polypeptide comprising a C-terminal domain of Cardiac Myosin binding protein C (MYBPC3).
A method of treating a disorder associated with abnormal ryanodine receptor type 2 (RYR2) function, the method comprising administering to a subject in need thereof an effective amount of a nucleic acid comprising a nucleotide sequence encoding a polypeptide comprising a C-terminal domain of Cardiac Myosin binding protein C (MYBPC3).
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(canceled)
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The method of claim 25, wherein administering the polypeptide or the nucleic acid reduces the excessive diastolic Ca2+ release in cardiomyocytes in the subject.
A method of treating arrhythmia, the method comprising administering to a subject in need thereof an effective amount of a recombinant adeno-associated virus (rAAV), wherein the rAAV comprises a capsid protein of serotype AAV9 and a nucleotide sequence encoding a polypeptide comprising a C-terminal domain of Cardiac Myosin binding protein C (MYBPC3).
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A recombinant adeno-associated virus (rAAV) comprising a capsid protein and a nucleotide sequence encoding a polypeptide comprising a C-terminal domain of Cardiac Myosin binding protein C (MYBPC3).
(canceled)
Dependencies are inferred from the claim text.