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首次直接植入体内的CRISPR治疗方法

CRISPR Treatment Inserted Directly into the Body for the First Time

A person with a genetic condition that causes blindness has become the first to receive a CRISPR–Cas9 gene therapy administered directly into their body.

一名由遗传条件导致失明的患者成为第一个接受CRISPR-Cas9基因治疗的患者。

The treatment is part of a landmark clinical trial to test the ability of CRISPR–Cas9 gene-editing techniques to remove mutations that cause a rare condition called Leber’s congenital amaurosis 10 (LCA10). No treatment is currently available for the disease, which is a leading cause of blindness in childhood.

这项治疗是一项具有里程碑意义的临床试验的一部分,目的是为了测试CRISPR-Cas9基因编辑技术去除突变的能力,这种突变会导致一种罕见的情况,即莱伯氏先天性黑蒙症(LCA10),而这种疾病是导致儿童失明的主要原因,但目前尚无治疗方法。

For the latest trial, the components of the gene-editing system – encoded in the genome of a virus—are injected directly into the eye, near photoreceptor cells. By contrast, previous CRISPR–Cas9 clinical trials have used the technique to edit the genomes of cells that have been removed from the body. The material is then infused back into the patient.

在最新的试验中基因编辑系统的组成部分——编码在病毒的基因组中——被直接注射到眼睛里,接近光感受器细胞;相比之下,以前的CRISPR-Cas9临床试验已经使用这种技术来编辑从体内移除的细胞的基因组,然后再把这些材料输回病人体内。

Read more at Scientific American

翻译:SGCS翻译组

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