Restoration of SERCA2a

October 28, 2010
The goal of the proposed treatment is to restore a key transporter SERCA2a which is deficient in heart failure to normal levels by gene therapy. Using standard cardiac angiography the gene for Serca2a, carried by a type 1 Adeno associated vector or AAV1, is injected antegrade down the coronary arteries using a slow perfusion. The AAV1 vector then passes through the capillaries and enters the myocardial space. AAV1 attaches to the surface receptors of the failing myocardial cells releasing the gene encoding for SERCA2a which then enters the nucleus. Messenger RNA is then produced encoding for the SERCA2a protein, which is then trafficked to the Sarcoplasmic Reticulum. This allows a restoration of SERCA2a to normal levels resulting in enhanced contraction of the cardiac cells and improved function of the ventricles and the whole heart.