CRISPR/Cas9技术在遗传性心脏疾病中的研究进展

CRISPR/Cas9 technology and its application in hereditary heart diseases

CRISPR/Cas9作为第三代基因编辑技术,能在分子水平上修改生物体的遗传信息,广泛应用于基因功能研究、模型构建以及基因治疗等领域,具有编辑效率高、操作简单、成本低、识别位点多样化等优点。遗传性心脏疾病是指通过基因显性遗传给下一代形成的心脏病,一直是与人类疾病相关发病率和死亡率的主要原因。本文就CRISPR/Cas9技术的发展历程、组成原理以及在遗传性心脏病的建模和治疗做详细阐述,并对CRISPR/Cas9技术的发展前景进行预见。

CRISPR/Cas9 as the third next-generation gene editing technology can modify the genetic information of organisms at the molecular level. It is widely used in the fields of gene function research, model construction and gene therapy. It has the advantages of high editing efficiency, simple operation, low cost, and diversified recognition sites. Inherited heart disease refers to heart disease formed through genetic dominant inheritance to the next generation. It has always been the main cause of morbidity and mortality related to human diseases. This article elaborates on the development history, composition principles, and the modeling and treatment of genetic heart disease of CRISPR/Cas9 technology, and predicts the development prospects of CRISPR/Cas9 technology.