The Impact of CRISPR/Cas9 Technology on Cardiac Research: From Disease Modelling to Therapeutic Approaches

Genome-editing technology has emerged as a powerful method that enables the generation of genetically modified cells and organisms necessary to elucidate gene function and mechanisms of human diseases. The clustered regularly interspaced short palindromic repeats- (CRISPR-) associated 9 (Cas9) syste...

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Main Authors: Benedetta M. Motta, Peter P. Pramstaller, Andrew A. Hicks, Alessandra Rossini
Format: Article
Language:English
Published: Wiley 2017-01-01
Series:Stem Cells International
Online Access:http://dx.doi.org/10.1155/2017/8960236
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author Benedetta M. Motta
Peter P. Pramstaller
Andrew A. Hicks
Alessandra Rossini
author_facet Benedetta M. Motta
Peter P. Pramstaller
Andrew A. Hicks
Alessandra Rossini
author_sort Benedetta M. Motta
collection DOAJ
description Genome-editing technology has emerged as a powerful method that enables the generation of genetically modified cells and organisms necessary to elucidate gene function and mechanisms of human diseases. The clustered regularly interspaced short palindromic repeats- (CRISPR-) associated 9 (Cas9) system has rapidly become one of the most popular approaches for genome editing in basic biomedical research over recent years because of its simplicity and adaptability. CRISPR/Cas9 genome editing has been used to correct DNA mutations ranging from a single base pair to large deletions in both in vitro and in vivo model systems. CRISPR/Cas9 has been used to increase the understanding of many aspects of cardiovascular disorders, including lipid metabolism, electrophysiology and genetic inheritance. The CRISPR/Cas9 technology has been proven to be effective in creating gene knockout (KO) or knockin in human cells and is particularly useful for editing induced pluripotent stem cells (iPSCs). Despite these progresses, some biological, technical, and ethical issues are limiting the therapeutic potential of genome editing in cardiovascular diseases. This review will focus on various applications of CRISPR/Cas9 genome editing in the cardiovascular field, for both disease research and the prospect of in vivo genome-editing therapies in the future.
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spelling doaj-art-c4eaaede5cf34864b3c1158e8571d4b72025-08-20T02:18:47ZengWileyStem Cells International1687-966X1687-96782017-01-01201710.1155/2017/89602368960236The Impact of CRISPR/Cas9 Technology on Cardiac Research: From Disease Modelling to Therapeutic ApproachesBenedetta M. Motta0Peter P. Pramstaller1Andrew A. Hicks2Alessandra Rossini3Institute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, ItalyInstitute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, ItalyInstitute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, ItalyInstitute for Biomedicine, Eurac Research, Affiliated Institute of the University of Lübeck, Bolzano, ItalyGenome-editing technology has emerged as a powerful method that enables the generation of genetically modified cells and organisms necessary to elucidate gene function and mechanisms of human diseases. The clustered regularly interspaced short palindromic repeats- (CRISPR-) associated 9 (Cas9) system has rapidly become one of the most popular approaches for genome editing in basic biomedical research over recent years because of its simplicity and adaptability. CRISPR/Cas9 genome editing has been used to correct DNA mutations ranging from a single base pair to large deletions in both in vitro and in vivo model systems. CRISPR/Cas9 has been used to increase the understanding of many aspects of cardiovascular disorders, including lipid metabolism, electrophysiology and genetic inheritance. The CRISPR/Cas9 technology has been proven to be effective in creating gene knockout (KO) or knockin in human cells and is particularly useful for editing induced pluripotent stem cells (iPSCs). Despite these progresses, some biological, technical, and ethical issues are limiting the therapeutic potential of genome editing in cardiovascular diseases. This review will focus on various applications of CRISPR/Cas9 genome editing in the cardiovascular field, for both disease research and the prospect of in vivo genome-editing therapies in the future.http://dx.doi.org/10.1155/2017/8960236
spellingShingle Benedetta M. Motta
Peter P. Pramstaller
Andrew A. Hicks
Alessandra Rossini
The Impact of CRISPR/Cas9 Technology on Cardiac Research: From Disease Modelling to Therapeutic Approaches
Stem Cells International
title The Impact of CRISPR/Cas9 Technology on Cardiac Research: From Disease Modelling to Therapeutic Approaches
title_full The Impact of CRISPR/Cas9 Technology on Cardiac Research: From Disease Modelling to Therapeutic Approaches
title_fullStr The Impact of CRISPR/Cas9 Technology on Cardiac Research: From Disease Modelling to Therapeutic Approaches
title_full_unstemmed The Impact of CRISPR/Cas9 Technology on Cardiac Research: From Disease Modelling to Therapeutic Approaches
title_short The Impact of CRISPR/Cas9 Technology on Cardiac Research: From Disease Modelling to Therapeutic Approaches
title_sort impact of crispr cas9 technology on cardiac research from disease modelling to therapeutic approaches
url http://dx.doi.org/10.1155/2017/8960236
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