Recent Progress in Genome Editing Approaches for Inherited Cardiovascular Diseases
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.
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.
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.
KeywordsGenome editing Base editing CRISPR/Cas9 Cardiovascular diseases
Compliance with Ethical Standards
Conflict of Interest
Balpreet Kaur, Isaac Perea Gil, and Ioannis Karakikes declare that they have no conflict of interest.
Human and Animal Rights and Informed Consent
This article does not contain any studies with human or animal subjects performed by any of the authors.
Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance
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