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Mount Sinai's Novel Gene Therapy Works to Reverse Heart Failure
Date:11/13/2013

, according to Dr. Hajjar, the time it took investigators to translate their basic laboratory findings to successful preclinical studies was very short.

“The key reason for this translational medicine speed is the outstanding infrastructure we have in the Cardiovascular Research Center at Mount Sinai, where we are able to replicate human heart failure models to test our novel gene therapies,” says Dr. Hajjar. “I think this is a really very unique example of rapid translation of a promising medical therapy from an initial discovery to pre-clinical trials.”

Other study investigators are all from Icahn School of Medicine at Mount Sinai. They include co-first authors Lisa Tilemann, MD, and Ahyoung Lee, PhD. Additional co-authors are Kiyotake Ishikawa, MD, Jaume Aguero, MD, Kleopatra Rapti, PhD, Carlos Santos-Gallego, MD, Erik Kohlbrenner, BS, Kenneth Fish, PhD, and Changwon Kho, PhD.

The study was supported by a National Institutes of Health grants (RO1 HL117505, HL119046, HL093183, P20HL100396), and a NHLBI Program of Excellence in Nanotechnology (PEN) Award (Contract # HHSN268201000045C).

Dr. Hajjar is the scientific cofounder of the company Celladon, which plans to develop AAV.SERCA2a gene therapy for the treatment of heart failure. All other authors declare no competing financial interests.

About the Mount Sinai Health System
The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven member hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services—from community-based facilities to tertiary and quaternary care.

The System includes approximately 6,600 primary and specialty care physicians, 12-minority-owned free-standing ambulatory surgery centers, over 45 ambulatory practices th
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