CRISPR-Cas9 in Cardiovascular Medicine: Unlocking New Potential for Treatment.

IF 5.2 2区 生物学 Q2 CELL BIOLOGY Cells Pub Date : 2025-01-17 DOI:10.3390/cells14020131
Klaudia Bonowicz, Dominika Jerka, Klaudia Piekarska, Janet Olagbaju, Laura Stapleton, Munirat Shobowale, Andrzej Bartosiński, Magdalena Łapot, Yidong Bai, Maciej Gagat
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Abstract

Cardiovascular diseases (CVDs) remain a significant global health challenge, with many current treatments addressing symptoms rather than the genetic roots of these conditions. The advent of CRISPR-Cas9 technology has revolutionized genome editing, offering a transformative approach to targeting disease-causing mutations directly. This article examines the potential of CRISPR-Cas9 in the treatment of various CVDs, including atherosclerosis, arrhythmias, cardiomyopathies, hypertension, and Duchenne muscular dystrophy (DMD). The technology's ability to correct single-gene mutations with high precision and efficiency positions it as a groundbreaking tool in cardiovascular therapy. Recent developments have extended the capabilities of CRISPR-Cas9 to include mitochondrial genome editing, a critical advancement for addressing mitochondrial dysfunctions often linked to cardiovascular disorders. Despite its promise, significant challenges remain, including off-target effects, ethical concerns, and limitations in delivery methods, which hinder its translation into clinical practice. This article also explores the ethical and regulatory considerations surrounding gene editing technologies, emphasizing the implications of somatic versus germline modifications. Future research efforts should aim to enhance the accuracy of CRISPR-Cas9, improve delivery systems for targeted tissues, and ensure the safety and efficacy of treatments in the long term. Overcoming these obstacles could enable CRISPR-Cas9 to not only treat but also potentially cure genetically driven cardiovascular diseases, heralding a new era in precision medicine for cardiovascular health.

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CRISPR-Cas9在心血管医学中的应用:释放新的治疗潜力
心血管疾病(cvd)仍然是一项重大的全球健康挑战,目前许多治疗方法针对的是症状,而不是这些疾病的遗传根源。CRISPR-Cas9技术的出现彻底改变了基因组编辑,提供了一种直接针对致病突变的变革性方法。本文探讨了CRISPR-Cas9在治疗各种心血管疾病中的潜力,包括动脉粥样硬化、心律失常、心肌病、高血压和杜氏肌营养不良症(DMD)。该技术以高精度和高效率纠正单基因突变的能力使其成为心血管治疗的开创性工具。最近的发展已经将CRISPR-Cas9的功能扩展到包括线粒体基因组编辑,这是解决通常与心血管疾病相关的线粒体功能障碍的关键进展。尽管它有希望,但仍然存在重大挑战,包括脱靶效应、伦理问题和给药方法的限制,这些都阻碍了它转化为临床实践。本文还探讨了围绕基因编辑技术的伦理和监管考虑,强调了体细胞与种系修饰的含义。未来的研究应致力于提高CRISPR-Cas9的准确性,改善靶向组织的递送系统,并确保治疗的长期安全性和有效性。克服这些障碍可以使CRISPR-Cas9不仅可以治疗而且有可能治愈遗传驱动的心血管疾病,预示着心血管健康精准医学的新时代。
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来源期刊
Cells
Cells Biochemistry, Genetics and Molecular Biology-Biochemistry, Genetics and Molecular Biology (all)
CiteScore
9.90
自引率
5.00%
发文量
3472
审稿时长
16 days
期刊介绍: Cells (ISSN 2073-4409) is an international, peer-reviewed open access journal which provides an advanced forum for studies related to cell biology, molecular biology and biophysics. It publishes reviews, research articles, communications and technical notes. Our aim is to encourage scientists to publish their experimental and theoretical results in as much detail as possible. There is no restriction on the length of the papers. Full experimental and/or methodical details must be provided.
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