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CRISPR/Cas9-mediated genome editing in nonhuman primates

机译:CRISPR / Cas9介导的非人类灵长类动物的基因组编辑

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Owing to their high similarity to humans, non-human primates (NHPs) provide an exceedingly suitable model for the study of human disease. In this Review, we summarize the history of transgenic NHP models and the progress of CRISPR/Cas9-mediated genome editing in NHPs, from the first proof-of-principle green fluorescent protein-expressing monkeys to sophisticated NHP models of human neurodegenerative disease that accurately phenocopy several complex disease features. We discuss not only the breakthroughs and advantages, but also the potential shortcomings of the application of the CRISPR/Cas9 system to NHPs that have emerged from the expanded understanding of this technology in recent years. Although off-target and mosaic mutations are the main concerns in CRISPR/Cas9-mediated NHP modeling, recent progress in genome editing techniques make it likely that these technical limitations will be overcome soon, bringing excellent prospects to human disease studies.
机译:由于它们与人类的高度相似性,非人类灵长类动物(NHP)为研究人类疾病提供了极为合适的模型。在这篇综述中,我们总结了转基因NHP模型的历史以及NHP中CRISPR / Cas9介导的基因组编辑的进展,从最早的原理性表达绿色荧光蛋白的猴子到精确的人类神经退行性疾病的复杂NHP模型,表型具有几种复杂的疾病特征。我们不仅讨论了突破和优势,而且还讨论了由于近年来对该技术的广泛了解而出现的将CRISPR / Cas9系统应用于NHP的潜在缺点。尽管脱靶和镶嵌突变是CRISPR / Cas9介导的NHP建模中的主要问题,但基因组编辑技术的最新进展使这些技术局限性有望很快被克服,为人类疾病研究带来了广阔的前景。

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