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Updated: Feb 3, 2026

Construction of Defined Human Engineered Cardiac Tissues to Study Mechanisms of Cardiac Cell Therapy
Published on: March 1, 2016
Human Cardiac Gene Therapy
Kiyotake Ishikawa1, Thomas Weber1, Roger J Hajjar1
1From the Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY.
Abstract:
In the past 10 years, there has been tremendous progress made in the field of gene therapy. Effective treatments of Leber congenital amaurosis, hemophilia, and spinal muscular atrophy have been largely based on the efficiency and safety of adeno-associated vectors. Myocardial gene therapy has been tested in patients with heart failure using adeno-associated vectors with no safety concerns but lacking clinical improvements. Cardiac gene therapy is adapting to the new developments in vectors, delivery systems, targets, and clinical end points and is poised for success in the near future.
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