CRISPR-Cas系统治疗心脏病的最新进展。

3区 生物学 Q2 Biochemistry, Genetics and Molecular Biology Progress in Molecular Biology and Translational Science Pub Date : 2025-01-01 Epub Date: 2024-08-31 DOI:10.1016/bs.pmbts.2024.08.004
Ingita Dey Munshi, Mansi Acharya, Sridip Mukherjee, Indra Mani
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引用次数: 0

摘要

CRISPR-Cas系统已成为基因研究领域的革命性工具,实现了高度精确的基因编辑,极大地推动了心血管科学领域的发展。本章全面概述了利用CRISPR-Cas技术研究和治疗心脏病的最新进展。它深入研究了CRISPR-Cas9在创建复杂心脏疾病(如肥厚性心肌病(HCM)、扩张性心肌病(DCM)和各种心律失常)的精确模型中的应用,这对于理解疾病机制和测试潜在治疗方法至关重要。研究人员还探索了基因编辑的治疗潜力,重点关注在脂质代谢和心血管健康中起关键作用的PCSK9和ANGPTL3等基因,为新疗法提供了有希望的途径。此外,研究人员还研究了CRISPR在心脏组织再生中的广泛应用,这可能会彻底改变受损心脏组织的修复。讨论了碱基编辑和引体编辑等前沿技术,强调了它们进一步完善遗传干预的潜力。讨论结束时,讨论了与高效、安全地递送CRISPR组件相关的挑战,同时也探讨了可能克服这些障碍的最新创新,为CRISPR技术在心血管医学中的未来方向提供了见解。
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Recent development in CRISPR-Cas systems for cardiac disease.

The CRISPR-Cas system has emerged as a revolutionary tool in genetic research, enabling highly precise gene editing and significantly advancing the field of cardiovascular science. This chapter provides a comprehensive overview of the latest developments in utilizing CRISPR-Cas technologies to investigate and treat heart diseases. It delves into the application of CRISPR-Cas9 for creating accurate models of complex cardiac conditions, such as hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy (DCM), and various arrhythmias, which are essential for understanding disease mechanisms and testing potential therapies. The therapeutic potential of gene editing is also explored, with a focus on genes like PCSK9 and ANGPTL3 that play critical roles in lipid metabolism and cardiovascular health, offering promising avenues for new treatments. Furthermore, the expanding applications of CRISPR in heart tissue regeneration are examined, which could revolutionize the repair of damaged heart tissue. Cutting-edge techniques such as base editing and prime editing are discussed, highlighting their potential to further refine genetic interventions. The discussion concludes by addressing the challenges associated with delivering CRISPR components efficiently and safely, while also exploring recent innovations that may overcome these hurdles, providing insights into the future directions of CRISPR technology in cardiovascular medicine.

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来源期刊
CiteScore
5.00
自引率
0.00%
发文量
110
审稿时长
4-8 weeks
期刊介绍: Progress in Molecular Biology and Translational Science (PMBTS) provides in-depth reviews on topics of exceptional scientific importance. If today you read an Article or Letter in Nature or a Research Article or Report in Science reporting findings of exceptional importance, you likely will find comprehensive coverage of that research area in a future PMBTS volume.
期刊最新文献
Advances in applications of the CRISPR/Cas9 system for respiratory diseases. Advances in CRISPR-Cas systems for kidney diseases. CRISPR challenges in clinical developments. Current approaches in CRISPR-Cas system for metabolic disorder. Current approaches in CRISPR-Cas systems for diabetes.
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