利用诱导多能干细胞和Crispr/Cas9构建更好的肥厚性心肌病模型

Zhe Han
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摘要

肥厚性心肌病(HCM)是导致美国年轻人心脏性猝死最多的一种心脏疾病,这一问题至今仍未得到解决。在本文中,我们将重新介绍HCM,并讨论可用于治疗它的方法。许多其他的研究,包括动物模型;诱导多能干细胞;CRISPR/Cas9也会被提及,以便更好地了解这种疾病。治疗HCM有许多有用的技术。其中一个有用的机制是iPSCs,它可以帮助重新编程患者的体细胞,将它们分化成研究所需的细胞。由于不同的背景问题,随后将添加CRISPR/ cas9来生成等基因iPSCs。结合这两种机制有助于比较HCM患者的iPSCs和对照iPSCs,从而为HCM的发病背景提供更多信息,进一步帮助HCM的治疗过程。
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Generate Better Hypertrophic Cardiomyopathy Model Using Induced Pluripotent Stem Cells and Crispr/Cas9
Hypertrophic cardiomyopathy (HCM) has been a heart disease causing the most sudden cardiac death among young people in US, and this problem still remains unsolved. In this paper, we will reintroduce HCM and discuss the methods that could be used to treat it. Many other studies, involving animal models; Induced pluripotent stem cells (iPSCs); CRISPR/Cas9, would also be mentioned to understand the disease better. Many techniques are useful in curing HCM. One of the helpful mechanisms would be iPSCs, which helps reprogramming a patient's somatic cells, differentiating them into cells needed for the study. Due to different background issues, CRISPR/Cas 9 would then be added to generate isogenic iPSCs. Combining both mechanisms would help in comparing patient's HCM iPSCs and control iPSCs, which provides us more information about pathogenic background of HCM and further assists in the process of curing HCM.
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