英国首次用基因编辑治好白血病女孩

A treatment that uses “molecular¹ scissors” to edit genes² has been used for the first time by UK medics successfully to treat a one-year-old girl with an “incurable³” form of leukaemia.

英国医生第一次依靠使用“分子剪刀”修改基因的疗法，成功地治愈了一名患有“无法治愈的”白血病的1岁女孩。

The case at Great Ormond Street Hospital in London involved the creation of “designer immune cells” programmed to hunt and kill the disease. The girl, called Layla, is now cancer free and doing well, according to the hospital.

伦敦大奥蒙德街医院(Great Ormond Street Hospital)的这一病例，生成经过设计的“人工培育的免疫细胞”去捕捉并治疗疾病。医院称，这个名叫“莱拉”(Layla)的女孩目前摆脱了癌症，身体状况很不错。

The breakthrough will add to excitement over the fast-emerging field of gene-editing — a type of genetic⁴ engineering in which DNA⁵ is inserted, replaced or removed from genes to fix faults or fight disease.

这次突破将令人对基因编辑这一快速发展的新兴领域感到更加兴奋。基因编辑是基因工程的一种，向基因中插入DNA，替换或取下其中的DNA，从而修复缺陷或战胜疾病。

Gene-editing has caused controversy⁶ because of its potential to be used in ethically⁷ dubious⁸ ways, such as the creation of “designer babies”. However, the London success helps show why many scientists are so enthusiastic⁹ about the technology.

基因编辑引发了争议，因为它可能导致令人产生道德疑虑的用法，比如生成“设计婴儿”。然而，这家伦敦医院的成功也证明，为何很多科学家那么热衷于这项技术。

The so-called UCART19 cells used at Great Ormond Street had shown promise in animals but had never been used in humans before they were administered as a last-ditch attempt to save Layla after other drugs failed.

大奥蒙德街医院使用的所谓UCART19细胞在动物身上已显出良好前景，但除了此次为救活莱拉而做的最后努力之前，该细胞从未应用于人体。此前在莱拉身上使用的其他药物全部失败了。

The treatment, developed by a French biotech company called Cellectis, consisted of 1ml of UCART19 cells injected into Layla’s bloodstream. After several weeks it was clear the leukaemia cells were disappearing.

这一疗法是由法国生物科技公司Cellectis开发出来的，把1毫升的UCART19细胞注入了莱拉的血流中。几周后，白血病细胞明显开始消失。

The treatment is similar to cancer therapies in development using modified T-cells from companies including Novartis of Switzerland and Juno and Kite of the US. However, whereas others use cells extracted from the patient’s own blood, UCART19 cells come from healthy donors¹¹.

该疗法类似于瑞士的诺华制药(Novartis)以及美国的Juno和Kite等公司开发的使用修饰T细胞治疗癌症的办法。然而，其他疗法使用从病人自身血液中提取的细胞，而UCART19细胞则来自于健康的捐献者。

This overcomes the problem of many leukaemia patients not having enough healthy T-cells — a type of white blood cell that fights off disease — to be harvested after chemotherapy.

这就克服了从化疗后的许多白血病患者体内难以提取到足量健康T细胞的难题。T细胞是一种能够对抗疾病的白血细胞。

A further possible advantage is that donor¹⁰ cells can be mass produced for use in any patient, making them potentially more affordable¹² than rival treatments that must be personalised for each individual. High costs are viewed as one of the biggest drawbacks of the so-called CAR-T therapies under development by Novartis, Juno and Kite.

一个更有可能的优势在于，捐赠细胞可以大规模生产，并用于任何患者，与因患者而异的其他疗法相比，在成本上更低。诺华制药、Juno和Kite正在研制中的所谓CAR-T疗法的最大缺点之一，就是被认为价格太高。