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Genome Editing Redefines Precision Medicine in the Cardiovascular Field

机译:基因组编辑重新定义了心血管领域的精密医学

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Genome editing is a powerful tool to study the function of specific genes and proteins important for development or disease. Recent technologies, especially CRISPR/Cas9 which is characterized by convenient handling and high precision, revolutionized the field of genome editing. Such tools have enormous potential for basic science as well as for regenerative medicine. Nevertheless, there are still several hurdles that have to be overcome, but patient-tailored therapies, termed precision medicine, seem to be within reach. In this review, we focus on the achievements and limitations of genome editing in the cardiovascular field. We explore different areas of cardiac research and highlight the most important developments (1) the potential of genome editing in human pluripotent stem cells in basic research for disease modelling, drug screening, or reprogramming approaches and (2) the potential and remaining challenges of genome editing for regenerative therapies. Finally, we discuss social and ethical implications of these new technologies.
机译:基因组编辑是研究对发育或疾病重要的特定基因和蛋白质功能的强大工具。最近的技术,特别是CRISPR / Cas9,以方便处理和高精度为特征,彻底改变了基因组编辑领域。这样的工具对于基础科学和再生医学具有巨大的潜力。尽管如此,仍然有几个障碍需要克服,但是患者定制的疗法,称为精密医学,似乎可以实现。在这篇综述中,我们关注于心血管领域中基因组编辑的成就和局限性。我们探索心脏研究的不同领域,并强调最重要的进展(1)在人类多能干细胞中进行疾病建模,药物筛选或重编程方法的基础研究中基因组编辑的潜力,以及(2)基因组的潜在挑战和尚存的挑战编辑再生疗法。最后,我们讨论这些新技术的社会和道德含义。

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