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Emerging Science for the Clinician
August 2017

The Hope and Hype of CRISPR-Cas9 Genome Editing: A Review

Author Affiliations
  • 1Cardiovascular Institute, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia
  • 2Department of Genetics, Perelman School of Medicine, University of Pennsylvania, Philadelphia
JAMA Cardiol. 2017;2(8):914-919. doi:10.1001/jamacardio.2017.1713

Importance  Clustered regularly interspaced short palindromic repeats (CRISPR) CRISPR-associated protein 9 (CRISPR-Cas9) has garnered a great degree of attention since its first reported uses in mammalian cells in early 2013 due to its perceived impact with respect to potential research applications and, especially, therapeutic applications.

Observations  CRISPR-Cas9 is being widely used in the laboratory and has greatly improved the ability to generate genetically modified animal models of human diseases. First steps have been reported with respect to proof-of-concept cardiovascular therapeutic applications in mouse models—which might eventually lead to new treatments for patients—as well as modification of human embryos, which raises a host of social, ethical, and legal questions.

Conclusions and Relevance  CRISPR-Cas9 is already having a substantial effect on cardiovascular basic science research. Although the first steps toward potential therapies have been taken, there are substantial obstacles that will need to be overcome if CRISPR-Cas9 is to be used in the practice of cardiovascular medicine.

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