Purpose of Review This review describes the recent progress in nuclease-based therapeutic applications for inherited heart diseases in vitro, highlights the development of the most recent genome editing technologies and discusses the associated challenges for clinical translation. Recent Findings Inherited cardiovascular disorders are passed from generation to generation. Over the past decade, considerable progress has been made in understanding the genetic basis of inherited heart diseases. The timely emergence of genome editing technologies using engineered programmable nucleases has revolutionized the basic research of inherited cardiovascular diseases and holds great promise for the development of targeted therapies. Summary The genome editing toolbox is rapidly expanding, and new tools have been recently added that significantly expand the capabilities of engineered nucleases. Newer classes of versatile engineered nucleases, such as the “base editors,” have been recently developed, offering the potential for efficient and precise therapeutic manipulation of the human genome. . . . Keywords Genome editing Base editing CRISPR/Cas9 Cardiovascular diseases Introduction that affect the electric system of the heart, causing arrhyth- mias. Clinical therapies for these inherited diseases focus on Cardiovascular diseases are the leading cause of death world- disease management without addressing the underlying genet- wide [1, 2]. According to
Current Cardiology Reports – Springer Journals
Published: Jun 2, 2018
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