哈佛大学将开始对精子进行基因编辑

导读

近期中国的一项实验声称已经创造出一对双胞胎女孩，她们的基因发生了改变，以保护她们免受艾滋病毒感染。在围绕这项实验的喧嚣中，有一些事情值得了解ーー改善下一代人类状况的研究也正在美国发生。尽管在中国爆发了一场关于基因改造婴儿的激烈辩论，但美国的一些科学家希望未来的孩子身体健康状况能够通过这种方法得到改善。

 正文

Despite CRISPR baby controversy, Harvard University will begin gene-editing sperm

尽管CRISPR 婴儿有争议，哈佛大学将开始对精子进行基因编辑

Even as a furious debate broke out in China over gene-edited babies, some scientists in the US are also hoping to improve tomorrow’s children.

尽管中国爆发了一场关于基因改造婴儿的激烈辩论，但美国的一些科学家也希望未来的孩子能得到改善

In the wild uproar around an experiment in China that claimed to have created twin girls whose genes were altered to protect them from HIV, there’s something worth knowing—research to improve the next generation of humans is happening in the US, too.

中国的一项实验声称已经创造出一对双胞胎女孩，她们的基因发生了改变，以保护她们免受艾滋病毒感染。在围绕这项实验的喧嚣中，有一些事情值得了解ーー改善下一代人类状况的研究也正在美国发生。

In fact, it’s about to happen at Harvard University.

事实上，这将发生在哈佛大学

At the school’s Stem Cell Institute, IVF doctor and scientist Werner Neuhausser says he plans to begin using CRISPR, the gene-editing tool, to change the DNA code insidesperm cells. The objective: to show whether it is possible to create IVF babies with a greatly reduced risk of Alzheimer’s disease later in life.

在学校的干细胞研究所，体外受精医生兼科学家 Werner neuhasser 说，他计划开始使用基因编辑工具 CRISPR 来改变精子细胞内的 DNA 编码。 研究目标: 证明是否有可能创造出能够大大降低老年痴呆症的风险的体外婴儿。

To be clear, there are no embryos involved—no attempt to make a baby. Not yet. Instead, the researchers are practicing how to change the DNA in sperm collected from Boston IVF, a large national fertility-clinic network. This is still very basic, and unpublished, research.

需要明确的是，这里没有涉及到胚胎，也没有试图制造一个婴儿。相反，研究人员正在练习如何改变从波士顿试管婴儿收集的精子中的DNA，这是一个庞大的国家生育诊所网络。 这仍然是一个非常基础的且尚未发表的研究。

Yet in its purpose the project is similar to the work undertaken in China and raises the same fundamental question: does society want children with genes tailored to prevent disease?

然而，该项目的目的与中国所做的类似，并且提出了同样且根本的问题: 社会是否希望儿童携带预防疾病的基因？

Since Sunday, when the CRISPR babiesclaims was made public,medicalbodies and experts have ferociously condemnedHe Jiankui, the Chinese scientist responsible.

There isevidencehis experiments—now halted—were carried forward in an unethical, deceptive manner that may have endangered the children he created. China’s vice minister of science and technology, Xu Nanping, said the effort “crossed the   line   of  moralityand ethics and was shocking and unacceptable.”

自上周日 CRISPR 编辑婴儿事件公布以来，医疗机构和专家对负责的中国科学家贺建奎进行了猛烈的谴责。 有证据表明，他现在已经被停止的实验是以一种不道德的欺骗方式进行的，这可能危及他所创造的孩子。 中国科技部副部长徐南平表示，这种行为"越过了道德伦理的底线，令人震惊和不可接受"

Amid the condemnation, though, it was easy to lose track of what the key experts were saying. Technology to alter heredity is for real. It is improving very quickly, it has features that will make it safe, and much wider exploratory use to create  children  could be justified soon. 然而，在一片谴责声中，人们很容易忘记关键专家的言论。 改变遗传的技术是真实的。它的进步非常迅速，它有能保证它安全的特点，更广泛的探索性使用去创造孩子（的做法）可能很快就会合理。

That was the message delivered at a gene-editing summit in Hong Kong on Wednesday, November 28, by Harvard Medical School dean George Daley, just ahead of He’s own  dramaticappearanceon the stage (seevideostarting at 1:15:30).这是哈佛医学院(Harvard Medical School)院长乔治•戴利(George Daley)于11月28日周三在香港举行的基因编辑峰会上传递的信息。

VF doctor and bench scientist Werner Neuhausser is exploring applicatio-ns of gene editing,

Astounding some listeners, the Harvard doctor and stem-cellresearcher didn’t condemn He but instead characterized the Chinese actions as a wrong turn on the right path . “The fact that it is possible that the first instance of human germ-line editing came forward as a misstep should in no way lead us to stick our heads in the sand,” Daley said. “It’s time to ... start outlining what an actual pathway for clinical translation would be.令一些听众感到震惊的是，这位哈佛医生和干细胞研究人员并没有谴责贺建奎，而是将中国的行为描述为在正确的道路上转错弯。戴利说:"第一次人类生殖细胞系编辑的出现可能是一个失误，这一事实绝不应该让我们把头埋在沙子里。"。 "是时候... ... 开始描述临床转化的实际途径了。"

By germ-line editing, Daley means editing sperm, eggs, or em-bryos—anything that, if you alter its DNA, could convey the change to future generations. While other voices demanded a ban on germ-line editing, Daley and the other members of the summit’s leadership defended it. Their final statement said medicine’s daring and troubled project to modify humans in an IVF dish should move forward.通过生殖细胞系编辑，戴利的意思是编辑精子、卵子或胚胎就意味着如果你改变了其中任何一个的DNA，就可能将这种改变遗传给后代。 当其他声音要求禁止生殖线编辑时，戴利和峰会的其他领导人为之辩护。 他们的最终声明说，在体外受精的过程中，医学界改造人类这种大胆而棘手项目应该向前推进。

“It’s absolutely clear this is a transformative scientific technology with the power for great medical use.” Daley said. "毫无疑问，这是一项革命性的科学技术，具有巨大的医疗用途。" 戴利说。

Harvard project

哈佛项目

Germ-line editing could be used, Daley said—and potentially should be used—to shape the health of tomorrow’s kids. By editing germ cells, it will be possible to remove mutations that cause childhood cancer or cystic fibrosis. Other genetic edits could endow children with protection against common diseases.     On Daley’s list of potentially acceptable genes to edit was CCR5, the very gene that He altered in the twins. 戴利表示，编辑生殖细胞系可以被使用，且有可能被用来塑造未来孩子们的健康。 通过编辑生殖细胞，可以去除引起儿童癌症或囊性纤维化的突变。 其他基因编辑可以赋予儿童保护力抵抗常见的疾病。 在戴利编辑列表上的潜在可接受基因是 CCR5，他在双胞胎身上改变了这个基因。

At Harvard, Neuhausser says he and a research fellow, Denis Vaughan, will in the next few weeks begin editing sperm to change a gene called ApoE, which is strongly linked to Alzheimer’s risk. A person who inherits two copies of the high-risk version of the gene has about a 60% lifetime risk of getting Alzheimer’s.在哈佛大学，诺豪瑟说，他和一个研究员，丹尼斯沃恩，将在未来几周内开始编辑精子来改变一个名为 ApoE 的基因，这个基因与患阿尔茨海默病的风险密切相关。一个人如果继承了两个高风险版本的基因，那么他一生中患老年痴呆症的风险就是60% 。

Neuhausser, an Austrian fertility doctor who came to the US to do his research and practice at Boston IVF, predicts that in not so many years, embryos will be deeply analyzed, selected, and in some cases altered with CRISPR before they are used to create a pregnancy. “In the future, people will go to clinics and get their genomes tested, and have the healthiest baby they can have,” he says. “I think the whole field will switch from fertility to disease prophylaxis”—preventing illness. 来自奥地利的生育医生 Neuhasser 在 Boston IVF 来到美国进行他们的研究和实践，他预测在不到几年的时间里，胚胎将被深入分析、选择，在某些情况下，在 CRISPR 被改变之后，它们将被用于怀孕。 他说:"将来，人们会去诊所检测自己的基因，生出最健康的宝宝。"。 "我认为整个领域将从生育转向疾病预防"ーー预防疾病。

To alter the DNA inside sperm cells, the team is using a clevernew version of CRISPR called base editing, developed by ano--ther Harvard scientist, David Liu. Instead of breaking open the double helix, base editing can flip a single genetic letter from, say, G to A. One such molecular tweak is enough to turn a the riskiest version of the ApoE gene into the least risky. 为了改变精子细胞内的 DNA，研究小组使用了一种叫做碱基编辑的新型 CRISPR 技术，这种技术是由另一位哈佛科学家 David Liu 开发的。 基础编辑可以把一个单独的遗传字母，比如说，从 g 变成 a，而不是打破双螺旋。一个这样的分子变化就足以把最危险的 ApoE 基因变成风险最小的。

“It’s one letter, G to A. You take it from risk to non-risk,” says Neuhausser."这是一个字母，g 到 a。 你把它从风险变为无风险,"纽豪瑟说。

Backlash

反弹

“You know what is risky? Speaking to journalists these days about how we are altering the germ line. But We haven’t ever shied from telling me what’s going on in the lab. ”“你知道什么是冒险吗？ 这些天跟记者谈论我们是如何改变生殖细胞系的。 但是我们从不回避告诉我的实验室里发生了什么。”

And lack of transparency is one reason the Chinese experiment is so troubling. It was done secretly, and ignored China’s rules forbidding such work. “The problem is that it’s going to make things much harder for everyone else following the rules if you jump so far ahead without proper approvals,” says Neuhausser. “That is the main concern. I don’t think the research is controversial, but everyone agrees it should be kept away from patients for now.” 缺乏透明度是中国的实验令人如此不安的原因之一。 这项实验是秘密进行的，无视了中国禁止这种做法的规定。 "问题在于，如果你在没有得到适当批准的情况下就大踏步前进，那么其他人要遵守规则就会变得更加困难。"。 "Neuhausser说道。这是最主要的问题。 我不认为这项研究有什么争议，但大家都认为目前应该让它远离病人。"

Harvard Medical School dean George Q. Daley is defending a new form of medicine in which children's DNA is changed.   

For now, ApoE is a toy example, one to try in the lab to test the technology and its potential. It’s not certain yet whether changing this gene would alter a child’s risk of Alzheimer’s later in life. Despite very strong links to the brain disease, there is no rock-solid proof that ApoE is a cause. “It’s one of the main risk factors for Alzheimer’s, although no one has shown causality,” says Neuhausser. “The point is to show the principle.”现在，ApoE 只是一个玩具例子，一个在实验室测试这项技术及其潜力的尝试。 目前还不确定改变这种基因是否会改变儿童日后患阿尔茨海默病的风险。 尽管 ApoE 与大脑疾病有很强的联系，但是并没有确凿的证据证明 ApoE 是致病因素。 "这是老年痴呆症的主要危险因素之一，尽管还没有人表明因果关系,"neuhasser 说。 "关键是要展示原则。"

But slashing a newborn’s lifelong risk of Alzheimer’s would be a huge deal. So would the ability to fix mutations that cause Lou Gehrig’s disease, another illness the team is looking into. Neuhausser expects it will eventually be routine to improve the DNA of sperm or embryos—and the people they turn into—in   fundamental ways.但是，降低新生儿终身患阿尔茨海默氏症的风险将是一件大事。 修复导致肌萎缩性侧索硬化症的突变的能力也是如此，这是研究小组正在研究的另一种疾病。 纽豪瑟预计，从根本上来说，改善精子或胚胎(以及它们变成的人)的 DNA，最终将成为常规。

Saving the species

拯救物种

With Boston IVF, Neuhausser has been carrying out a survey of doctors and hundreds of patients about what they think. “For treating or preventing disease, pretty much everyone agrees,” he says—that is, they are for it.与波士顿体外受精公司合作，纽豪瑟一直在对医生和数百名患者进行调查，了解他们的想法。 "对于治疗或预防疾病，几乎每个人都同意,"他说ーー也就是说，他们支持这种做法。

People do draw the line at things like increasing height or changing eye color, with only a tiny percentage thinking it’s a good idea. Neuhausser admits someone might do that too, eventually. “Like any technology, there will be misuses,” he says. “But it’s important that we return to a rational approach, recognizing that this has huge potential and huge risks. The problem is that when people get scared, things get shut down. That is why people are nervous about He—he’s hurting everyone else.”人们确实会在增加身高或改变眼睛颜色这样的事情上划清界限，只有很小一部分人认为这是个好主意。 纽豪瑟承认，最终也会有人这样做。 他表示:"与任何技术一样，都会出现误用。"。 "但重要的是，我们要回归理性的方式，承认这种做法有巨大的潜力和巨大的风险。 问题是，当人们感到害怕的时候，事情就被终止了。 这就是为什么人们对他感到紧张ーー他在伤害其他人。"

To anyone who wants to end this line of research, the Harvard doctors have one last ace to play. They say germ-line editing might be an important technology to have for civilization’s sake.对于任何想要结束这项研究的人来说，哈佛医生还有最后一张王牌可以使用。 他们说，为了文明的缘故，生殖细胞基因编辑可能是一项重要的技术。

What if a new killer virus arises and sweeps the world? Maybe there will be no vaccine but some people will be able to resist  it thanks to their genes, as some fared better with the Black Death in medieval times. Wouldn’t we want to then give the genetic antidote to all members of the next generation?如果一个新的杀手病毒出现并横扫世界会怎么样？ 也许没有疫苗，但有些人能够抵抗，这要归功于他们的基因，因为有些人在中世纪的黑死病中表现得更好。 难道我们不想给下一代所有成员遗传解毒剂？

“This is a technology that could save the species, potentially,” says Neuhausser. In his speech in Hong Kong, Daley also referenced the potential defense against future disease.诺豪瑟说:"这项技术有可能拯救这个物种。"。 在香港的演讲中，戴利也提到了对抗未来疾病的可能性。

“We as a species need to maintain the flexibility to face future threats, to take control of our heredity,” he said."我们作为一个物种需要保持灵活性以面对未来的威胁，来控制我们的遗传,"他说。

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