近日，一支中國(guó)的科學(xué)家團(tuán)隊(duì)首次利用CRISPR-Cas9基因編輯技術(shù),，將編輯過(guò)的細(xì)胞注入了一名病人體內(nèi),。這也是該技術(shù)在全球首次被直接應(yīng)用于人體,，在基因研究領(lǐng)域具有里程碑式的意義。

據(jù)《自然》雜志報(bào)道,，10月28日,，四川大學(xué)的研究人員們?cè)谒拇ù髮W(xué)華西醫(yī)院將經(jīng)過(guò)基因編輯的細(xì)胞注入到了一名參與臨床試驗(yàn)的肺癌患者體內(nèi)。這支團(tuán)隊(duì)是由腫瘤學(xué)家盧鈾醫(yī)生領(lǐng)導(dǎo)的,。

這種旨在治療癌癥的CRISPR基因編輯技術(shù)需要進(jìn)行一系列的分子切割,，引導(dǎo)Cas9分子“敲”掉免疫細(xì)胞內(nèi)可能滋生癌癥的致病基因。然后這些被編輯過(guò)的細(xì)胞會(huì)被重新放回患者體內(nèi),，用以攻擊惡性腫瘤,。

此次人體試驗(yàn)原本計(jì)劃于今年八月進(jìn)行，由于培養(yǎng)被編輯的細(xì)胞所需要的時(shí)間超出了最初的預(yù)期,，因而一直拖到十月底才得以實(shí)施,。不過(guò)中國(guó)人在CRISPR技術(shù)上的試驗(yàn)步伐依然遠(yuǎn)遠(yuǎn)走在了美國(guó)人的前頭。美國(guó)的首次CRISPR人體試驗(yàn)?zāi)壳斑€處于計(jì)劃階段,，雖然今年年初,，美國(guó)的研究人員已經(jīng)獲準(zhǔn)進(jìn)行此項(xiàng)試驗(yàn)（并且獲得了科技界的億萬(wàn)富翁肖恩·帕克的投資），但估計(jì)一直要等到2017年年初,，這項(xiàng)試驗(yàn)才會(huì)在賓西法尼亞大學(xué)進(jìn)行,。

癌癥免疫學(xué)家卡爾·瓊恩博士對(duì)《自然》雜志表示，中國(guó)科學(xué)家率先實(shí)施此項(xiàng)技術(shù)的臨床測(cè)試,，很可能會(huì)引發(fā)中美兩國(guó)在此項(xiàng)技術(shù)上的良性競(jìng)爭(zhēng),。

“我認(rèn)為在生物醫(yī)學(xué)領(lǐng)域上將爆發(fā)一場(chǎng)新的‘美蘇太空競(jìng)賽’，不過(guò)這次的競(jìng)爭(zhēng)雙方將是中國(guó)和美國(guó),。不過(guò)競(jìng)爭(zhēng)也是很重要的，因?yàn)樗ǔＳ兄谔岣咦罱K產(chǎn)品的質(zhì)量,?！?/p>

中國(guó)科學(xué)家的此次試驗(yàn)將在10名患者身上進(jìn)行，被試者每人將接受2至4次的編輯細(xì)胞注射,。該研究的重點(diǎn)在于確定這項(xiàng)基因療法的安全性,，以及它是否會(huì)產(chǎn)生令人無(wú)法接受的副作用。（財(cái)富中文網(wǎng)）

譯者：樸成奎

In a landmark for genomic research, a team of Chinese scientists has injected cells modified with the groundbreaking CRISPR-Cas9 gene-editing technology into a patient. It’s the first known time the technique has actually been deployed in a human.

Researchers from the Sichuan University in Chengdu inserted the re-engineered cells into a lung cancer patient participating in a clinical trial at the West China Hospital on October 28th, according to Nature. The team is being led by oncologist Dr. Lu You.

Cancer-focused CRISPR technology involves taking a set of molecular shears and the guiding molecule Cas9 in order to cut out unwanted genes in immune cells that may help proliferate cancers. These modified cells are then put back into patients in order to attack cancerous tumors.

The trial was actually supposed to launch back in August but was delayed because growing and culturing the genomically edited cells took longer than originally expected. Regardless, the study has begun well ahead of the first planned American CRISPR trials, which won regulatory clearance earlier this year (and are being funded by tech billionaire Sean Parker) but are unlikely to begin until 2017 at the University of Pennsylvania.

Still, the Chinese scientists’ first-to-the-clinic victory may help fuel positive rivalries in the U.S., scientist and cancer immunotherapy expert Dr. Carl June told Nature.

“I think this is going to trigger ‘Sputnik 2.0’, a biomedical duel on progress between China and the United States, which is important since competition usually improves the end product,” he said.

The Chinese trial will be conducted on ten patients who will receive anywhere from two to four injections of modified cells, and the study will mostly focus on how safe the treatments are and whether or not they have unacceptable side effects.