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Treatment of Dyslipidemia Using CRISPR/Cas9 Genome Editing

  • Genetics and Genomics (A. Marian, Section Editor)
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Current Atherosclerosis Reports Aims and scope Submit manuscript

Abstract

Purpose of Review

Clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated 9 (Cas9) has recently emerged as a top genome editing technology and has afforded investigators the ability to more easily study a number of diseases. This review discusses CRISPR/Cas9’s advantages and limitations and highlights a few recent reports on genome editing applications for alleviating dyslipidemia through disruption of proprotein convertase subtilisin/kexin type 9 (PCSK9).

Recent Findings

Targeting of mouse Pcsk9 using CRISPR/Cas9 technology has yielded promising results for lowering total cholesterol levels, and several recent findings are highlighted in this review. Reported on-target mutagenesis efficiency is as high as 90% with a subsequent 40% reduction of blood cholesterol levels in mice, highlighting the potential for use as a therapeutic in human patients.

Summary

The ability to characterize and treat diseases is becoming easier with the recent advances in genome editing technologies. In this review, we discuss how genome editing strategies can be of use for potential therapeutic applications.

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Authors and Affiliations

  1. Cardiovascular Institute, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA

    Alexandra C. Chadwick & Kiran Musunuru

  2. Department of Genetics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA

    Kiran Musunuru

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  1. Alexandra C. Chadwick
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Correspondence to Kiran Musunuru.

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Alexandra Chadwick and Kiran Musunuru declare they have no conflicts of interest.

Human and Animal Rights and Informed Consent

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. All applicable international, national, and/or institutional guidelines for the care and use of animals were followed. All procedures performed in studies involving animals were in accordance with the ethical standards of the institution or practice at which the studies were conducted.

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Chadwick, A.C., Musunuru, K. Treatment of Dyslipidemia Using CRISPR/Cas9 Genome Editing. Curr Atheroscler Rep 19, 32 (2017). https://doi.org/10.1007/s11883-017-0668-8

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