Serca2 therapeutic compositions and methods of use
AssigneesMount Sinai School of Medicine, Celladon Corporation
InventorsRoger J. Hajjar, Yoshiaki Kawase, Dennis Ladage, Krisztina Zsebo
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Abstract
The present invention provides methods for treating pulmonary hypertension in a subject by delivering a therapeutic adeno-associated virus (AAV)-SERCA2 composition to a subject in need thereof.
Claims26
A method for treating pulmonary hypertension in a subject comprising delivering a viral expression vector comprising a transgene into the subject, wherein the transgene modulates Ca+2 ion transport, and wherein expression of the transgene increases host cell function, thereby treating pulmonary hypertension.
A method for treating pulmonary hypertension in a subject comprising delivering a recombinant adeno-associated virus (AAV) virion to the subject, wherein the virion comprises an AAV vector comprising a transgene operably linked to control elements that direct expression of the transgene in a host cell, and wherein the expression of the transgene improves lung function.
A method for delivering a recombinant adeno-associated virus (rAAV) virion containing a transgene to a pulmonary muscle cell of a mammalian subject with pulmonary hypertension, comprising providing a recombinant AAV virion comprising a polynucleotide encoding a protein or RNAi capable of regulating a calcium cycling pathway of the pulmonary muscle cell, wherein the polynucleotide is operably linked to a control element capable of directing expression of the protein or RNAi;
- and delivering the rAAV virion directly into the pulmonary muscle cell of the subject, wherein the protein is expressed at a therapeutically effective level in the muscle cell.
Dependencies are inferred from the claim text.