SERCA Restoration Therapy for Heart Failure

The Sarco(Endo)plasmic reticulum Ca(2+) ATPase (SERCA) is instrumental for recycling cytosolic Ca(2+) into the lumen of the sarcoplasmic reticulum. Restoring adequate SERCA2a/b expression by pharmacological action or gene delivery has emerged as a new approach for the treatment of heart failure (HF).21 AAV1/SERCA2a, has been shown to be efficacious in clinical trials improving HF parameters in patients with advanced HF. It has been granted breakthrough therapy designation by FDA for reducing hospitalizations for HF in NYHA class III or IV chronic HF patients who are NAb negative.


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