University of Connecticut Health Center, Farmington, CT, USA.
The Jackson Laboratory for Genomic Medicine, Farmington, CT, USA.
Curr Cardiol Rep. 2024 Nov;26(11):1231-1240. doi: 10.1007/s11886-024-02125-3. Epub 2024 Sep 17.
Outline the growing suite of novel genome editing tools powered by CRISPR-Cas9 technology that are rapidly advancing towards the clinic for the treatment of cardiovascular disorders.
A diversity of new genome editors and modulators are being developed for therapies across myriad human diseases. Recent breakthroughs have improved the efficacy, safety, specificity, and delivery of CRISPR-mediated therapies that could impact heart disease in the next decade, though several challenges remain. Many iterations of the original CRISPR system have been developed seeking to leverage its vast therapeutic potential. As examples, nuclease-free editing, precision single-nucleotide editing, gene expression regulation, and epigenomic modifications are now feasible with the current CRISPR-mediated suite of enzymes. These emerging tools will be indispensable for the development of novel cardiovascular therapeutics as demonstrated by recent successes in both basic research laboratories and pre-clinical models. Here, we provide an overview of current and emerging CRISPR-mediated technologies as they pertain to the cardiovascular system, highlighting successful implementations and future challenges.
概述了一系列新兴的基于 CRISPR-Cas9 技术的新型基因组编辑工具，它们正在迅速推进心血管疾病的临床治疗。
为治疗各种人类疾病，正在开发多种新型基因组编辑器和调节剂。最近的突破提高了 CRISPR 介导疗法的疗效、安全性、特异性和递送效率，这可能会在未来十年内对心脏病产生影响，但仍存在一些挑战。许多原始 CRISPR 系统的迭代版本被开发出来，旨在利用其巨大的治疗潜力。例如，无核酸酶编辑、精确单核苷酸编辑、基因表达调控和表观遗传修饰现在都可以使用当前的 CRISPR 介导的酶实现。这些新兴工具对于新型心血管治疗药物的开发将是不可或缺的，正如最近在基础研究实验室和临床前模型中取得的成功所证明的那样。在这里，我们概述了与心血管系统相关的当前和新兴的 CRISPR 介导技术，强调了成功的应用和未来的挑战。
