Genome editing in cardiovascular diseases

Mani, Indra

doi:10.1016/bs.pmbts.2021.01.021

Cited by 65 publications

(3 citation statements)

References 121 publications

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“…[ 37 ] Recent evidence claims that this strategy is a novel treatment for several genetic disorders, including some cancers, neurodegenerative diseases, sickle cell anemia, DMD, viral infections, immune disorders, cystic fibrosis, and CVDs. [ 38 39 40 41 ] Various studies have supported this hypothesis that the combination of genome-wide association studies and CRISPR genome-engineering strategy could play an important role in the development of human personalized medicine. [ 41 ]…”

Section: Basic Studies and Clinical Findingsmentioning

confidence: 99%

A New Era of Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated Protein 9 Gene Editing Technology in Cardiovascular Diseases: Opportunities, Challenges, and Perspectives

Kumar,

Singh,

Kumar

et al. 2023

Heart Views

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Cardiovascular diseases (CVDs) remain major causes of global mortality in the world. Genetic approaches have succeeded in the discovery of the molecular basis of an increasing number of cardiac diseases. Genome-editing strategies are one of the most effective methods for assisting therapeutic approaches. Potential therapeutic methods of correcting disease-causing mutations or of knocking out specific genes as approaches for the prevention of CVDs have gained substantial attention using genome-editing techniques. Recently, the clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9) system has become the most widely used genome-editing technology in molecular biology due to its benefits such as simple design, high efficiency, good repeatability, short cycle, and cost-effectiveness. In the present review, we discuss the possibilities of applying the CRISPR/Cas9 genome-editing tool in the CVDs.

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Section: Basic Studies and Clinical Findingsmentioning

confidence: 99%

A New Era of Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated Protein 9 Gene Editing Technology in Cardiovascular Diseases: Opportunities, Challenges, and Perspectives

Kumar,

Singh,

Kumar

et al. 2023

Heart Views

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“…Most intriguing is the fact that the functions of most ncRNAs are still unknown [3,4], although they may be involved in the pathogenesis of a disease or considered as a potential therapeutic target or drug. The classical approaches to establishing the functions of individual molecules in biological systems are the inhibition of the target molecule, its exogenous administration or the modification of its structure [5][6][7]. The same approaches (overexpression, repression or sequence change) can be applied to study the functions and molecular partners of individual ncRNAs.…”

Section: Introductionmentioning

confidence: 99%

Advances and Obstacles in Using CRISPR/Cas9 Technology for Non-Coding RNA Gene Knockout in Human Mesenchymal Stromal Cells

Basalova,

Illarionova,

Skryabina

et al. 2023

ncRNA

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Non-coding RNA (ncRNAs) genes have attracted increasing attention in recent years due to their widespread involvement in physiological and pathological processes and regulatory networks. The study of the function and molecular partners of ncRNAs opens up opportunities for the early diagnosis and treatment of previously incurable diseases. However, the classical “loss-of-function” approach in ncRNA function analysis is challenged due to some specific issues. Here, we have studied the potency of two CRISPR/Cas9 variants, wild-type (SpCas9wt) and nickase (SpCas9D10A) programmable nucleases, for the editing of extended DNA sequences in human mesenchymal stromal cells (MSCs). Editing the genes of fibrosis-related hsa-miR-21-5p and hsa-miR-29c-3p, we have shown that a pair of SpCas9D10A molecules can effectively disrupt miRNA genes within the genomes of MSCs. This leads not only to a decrease in the level of knockout miRNA in MSCs and MSC-produced extracellular vesicles, but also to a change in cell physiology and the antifibrotic properties of the cell secretome. These changes correlate well with previously published data for the knockdown of certain miRNAs. The proposed approach can be used to knock out ncRNA genes within the genomes of MSCs or similar cell types in order to study their function in biological processes.

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“…A growing understanding of the genetics underlying cardiac arrhythmias has enabled new treatment possibilities including the use of cardiac genome editing [7]. Monogenetic diseases associated with cardiac arrhythmias include congenital long QT syndrome (LQTS), short QT interval syndrome, Brugada syndrome, catecholaminergic polymorphic ventricular tachycardia (CPVT), idiopathic ventricular fibrillation, Wolff-Parkinson-White syndrome, arrhythmogenic cardiomyopathy, and other cardiomyopathy syndromes associated with an increased risk of arrhythmias such as dilated cardiomyopathy (DCM), hypertrophic cardiomyopathy (HCM) and hereditary transthyretin amyloidosis (ATTR-CM) [8].…”

Section: Introductionmentioning

confidence: 99%

Genome Editing and Cardiac Arrhythmias

Moore

Copeland

et al. 2023

Cells

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This article reviews progress in the field of cardiac genome editing, in particular, its potential utility in treating cardiac arrhythmias. First, we discuss genome editing methods by which DNA can be disrupted, inserted, deleted, or corrected in cardiomyocytes. Second, we provide an overview of in vivo genome editing in preclinical models of heritable and acquired arrhythmias. Third, we discuss recent advancements in cardiac gene transfer, including delivery methods, gene expression optimization, and potential adverse effects associated with therapeutic somatic genome editing. While genome editing for cardiac arrhythmias is still in its infancy, this approach holds great promise, especially for inherited arrhythmia syndromes with a defined genetic defect.

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Genome editing in cardiovascular diseases

Cited by 65 publications

References 121 publications

A New Era of Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated Protein 9 Gene Editing Technology in Cardiovascular Diseases: Opportunities, Challenges, and Perspectives

A New Era of Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated Protein 9 Gene Editing Technology in Cardiovascular Diseases: Opportunities, Challenges, and Perspectives

Advances and Obstacles in Using CRISPR/Cas9 Technology for Non-Coding RNA Gene Knockout in Human Mesenchymal Stromal Cells

Genome Editing and Cardiac Arrhythmias

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