Gene editing of angiotensin for blood pressure management

Arterial hypertension has remained the world's leading cause of morbidity and mortality for more than 20 years. While early Genome-Wide Association Studies raised the hypothesis that a precision medicine approach could be implemented in the treatment of hypertension, the large number of single...

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Main Authors: Stefano Masi, Hermann Dalpiaz, Claudio Borghi
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:International Journal of Cardiology. Cardiovascular Risk and Prevention
Online Access:http://www.sciencedirect.com/science/article/pii/S2772487524000886
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author Stefano Masi
Hermann Dalpiaz
Claudio Borghi
author_facet Stefano Masi
Hermann Dalpiaz
Claudio Borghi
author_sort Stefano Masi
collection DOAJ
description Arterial hypertension has remained the world's leading cause of morbidity and mortality for more than 20 years. While early Genome-Wide Association Studies raised the hypothesis that a precision medicine approach could be implemented in the treatment of hypertension, the large number of single nucleotide polymorphisms that were found to be associated with blood pressure and their limited impact on the blood pressure values have initially hampered these expectations. With the development and refinement of gene-editing and RNA-based approaches allowing selective and organ-specific modulation of critical systems involved in blood pressure regulation, a renewed interest in genetic treatments for hypertension has emerged. The CRISPR-Cas9 system, antisense oligonucleotides (ASO) and small interfering RNA (siRNA) have been used to specifically target the hepatic angiotensinogen (AGT) production, with the scope of safely but effectively reducing the activation of the renin-angiotensin system, ultimately leading to an effective reduction of the blood pressure with extremely simplified treatment regimens that involve weekly, monthly or even once-in-life injection of the drugs. Among the various approaches, siRNA and ASO that reduce hepatic AGT production are in advanced development, with phase I and II clinical trials showing their safety and effectiveness. In the current manuscript, we review the mode of action of these new approaches to hypertension treatment, discussing the results of the clinical trials and their potential to revolutionize the management of hypertension.
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spelling doaj-art-6de6d2fe564e486785ab767db12ae4eb2025-08-20T01:54:15ZengElsevierInternational Journal of Cardiology. Cardiovascular Risk and Prevention2772-48752024-12-012320032310.1016/j.ijcrp.2024.200323Gene editing of angiotensin for blood pressure managementStefano Masi0Hermann Dalpiaz1Claudio Borghi2Department of Clinical and Experimental Medicine, University of Pisa, Italy; Corresponding author. Department of Clinical and Experimental Medicine University of Pisa, Via Roma, 67 560126, Pisa, Italy.Department of Clinical and Experimental Medicine, University of Pisa, ItalyHypertension and Cardiovascular Disease Research Center, Medical and Surgical Sciences Department, Alma Mater Studiorum University of Bologna, 40126, Bologna, Italy; Cardiovascular Medicine Unit, Heart-Chest-Vascular Department, IRCCS Azienda Ospedaliero-Universitaria di Bologna, 40126, Bologna, ItalyArterial hypertension has remained the world's leading cause of morbidity and mortality for more than 20 years. While early Genome-Wide Association Studies raised the hypothesis that a precision medicine approach could be implemented in the treatment of hypertension, the large number of single nucleotide polymorphisms that were found to be associated with blood pressure and their limited impact on the blood pressure values have initially hampered these expectations. With the development and refinement of gene-editing and RNA-based approaches allowing selective and organ-specific modulation of critical systems involved in blood pressure regulation, a renewed interest in genetic treatments for hypertension has emerged. The CRISPR-Cas9 system, antisense oligonucleotides (ASO) and small interfering RNA (siRNA) have been used to specifically target the hepatic angiotensinogen (AGT) production, with the scope of safely but effectively reducing the activation of the renin-angiotensin system, ultimately leading to an effective reduction of the blood pressure with extremely simplified treatment regimens that involve weekly, monthly or even once-in-life injection of the drugs. Among the various approaches, siRNA and ASO that reduce hepatic AGT production are in advanced development, with phase I and II clinical trials showing their safety and effectiveness. In the current manuscript, we review the mode of action of these new approaches to hypertension treatment, discussing the results of the clinical trials and their potential to revolutionize the management of hypertension.http://www.sciencedirect.com/science/article/pii/S2772487524000886
spellingShingle Stefano Masi
Hermann Dalpiaz
Claudio Borghi
Gene editing of angiotensin for blood pressure management
International Journal of Cardiology. Cardiovascular Risk and Prevention
title Gene editing of angiotensin for blood pressure management
title_full Gene editing of angiotensin for blood pressure management
title_fullStr Gene editing of angiotensin for blood pressure management
title_full_unstemmed Gene editing of angiotensin for blood pressure management
title_short Gene editing of angiotensin for blood pressure management
title_sort gene editing of angiotensin for blood pressure management
url http://www.sciencedirect.com/science/article/pii/S2772487524000886
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AT hermanndalpiaz geneeditingofangiotensinforbloodpressuremanagement
AT claudioborghi geneeditingofangiotensinforbloodpressuremanagement