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Recent Progress in Genome Editing Approaches for Inherited Cardiovascular Diseases

  • Balpreet Kaur
  • Isaac Perea-Gil
  • Ioannis Karakikes
Regenerative Medicine (SM Wu, Section Editor)
Part of the following topical collections:
  1. Topical Collection on Regenerative Medicine

Abstract

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 

Notes

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.

References

Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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Copyright information

© Springer Science+Business Media, LLC, part of Springer Nature 2018

Authors and Affiliations

  • Balpreet Kaur
    • 1
  • Isaac Perea-Gil
    • 1
    • 2
  • Ioannis Karakikes
    • 1
    • 2
  1. 1.Stanford Cardiovascular InstituteStanford University School of MedicineStanfordUSA
  2. 2.Department of Cardiothoracic SurgeryStanford University School of MedicineStanfordUSA

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