Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome
Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome
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DOI：
10.1038/s41591-018-0338-6
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发表时间：
2019-03-01
期刊：
影响因子：
82.9
通讯作者：
Lopez-Otin, Carlos
中科院分区：
文献类型：
--
作者：
Santiago-Fernandez, Olaya;Osorio, Fernando G.;Lopez-Otin, Carlos
基于CRISPR/Cas9的疗法在治疗遗传疾病方面具有相当大的前景。其中，由LMNA基因点突变引起的Hutchinson-Gilford早衰综合征是一个潜在的候选者。在这里，我们探索了一种基于CRISPR/Cas9的方法的有效性，该方法通过在LMNA基因中引入移码突变来逆转Hutchinson-Gilford早衰综合征细胞和小鼠中的几种改变。
CRISPR/Cas9-based therapies hold considerable promise for the treatment of genetic diseases. Among these, Hutchinson-Gilford progeria syndrome, caused by a point mutation in the LMNA gene, stands out as a potential candidate. Here, we explore the efficacy of a CRISPR/Cas9-based approach that reverts several alterations in Hutchinson-Gilford progeria syndrome cells and mice by introducing frameshift mutations in the LMNA gene.