2026年9月29日 下午1:00(美国东部时间) 罗布·斯坦(Rob Stein)Origin Genomics的创始人兼首席执行官凯茜·泰(Cathy Tie)正在为维持细胞生长的营养液补充养分,这家总部位于纽约的初创公司正利用这些细胞测试其修复缺陷基因的技术。
September 29, 2026 1:00 PM ET Rob Stein Cathy Tie, the founder and CEO of Origin Genomics, replenishes nutrients sustaining cells the New York City startup is using to test its technology for repairing defective genes.
麦肯齐·卡勒(Mackenzie Calle)/ NPR 为获取更多关于健康生活科学的报道,请订阅NPR的健康通讯。
Mackenzie Calle for NPR For stories on the science of healthy living, to NPR's Health newsletter.
凯茜·泰穿上了一件印有她名字和初创公司标志的白色实验室长袍:Origin Genomics “欢迎!”她一边说,一边带领NPR的一名记者、摄影师和摄像师参观位于纽约市的Origin实验室。出于安全原因,Origin的首席执行官泰要求NPR不要透露具体地点。“我们这就进实验室,”她说。
Cathy Tie slips on a white lab coat bearing her name and the logo of her startup: Origin Genomics "Welcome!" she says as she leads an NPR reporter, photographer and videographer on an exclusive tour of Origin's labs located in New York City. Tie, CEO of Origin, asked NPR to withhold the exact location for security reasons. "We'll head into the lab," she says.
一进去,泰就戴上一副蓝色橡胶手套,指着地板上一个看起来像巨大保温杯的灰色金属罐。
Once inside, Tie snaps on a pair of blue rubber gloves and points to a gray metal tank on the floor that looks like a giant thermos.
“这是一个存有液氮的低温储存罐,我们的胚胎就储存在这里,”她说。
"This is a cryogenic storage unit that has liquid nitrogen inside, which is where our embryos are stored," she says.
泰拧开黑色的盖子,液氮产生的缕缕白雾从罐中盘旋而出。泰轻轻抽出了一根长长的金属杆,其末端托着结霜的玻璃管。
Tie twists open the black lid, letting wisps of white fog from the liquid nitrogen swirl out from the tank. Tie gently slides out a long metal rod cradling frosted glass tubes at the end.
“大家可以看到,这里有四根管子,”泰说。“每根管子里最多可以存放10个胚胎。” 泰从Origin Genomics新实验室的低温储存罐中,取出了携带有致病突变的冷冻人类胚胎。
"So as you can see there's four tubes in here," Tie says. "Each tube can contain up to 10 embryos." Tie pulls out frozen human embryos carrying disease-causing mutations from a cryogenic storage unit at Origin Genomics new labs.
这些胚胎是由一些夫妇捐赠的,他们的家族世世代代饱受危及生命的遗传病的折磨。随后,捐赠了其中部分胚胎的一对夫妻向我们讲述了他们借助基因编辑孕育健康孩子的梦想。
These embryos were donated by couples whose families have been plagued for generations by life-threatening genetic disorders. Later, a wife and husband who donated some of those embryos tell us about their dream of having healthy children with the help of gene editing.
但首先,泰解释了Origin的科学家如何从胚胎中提取干细胞,以测试该公司实验性的基因编辑工具,从而确定哪种分子工具能够安全地矫正基因缺陷,防止后代遭受同样的命运。
But first, Tie explains how Origin's scientists extract stem cells from the embryos to test the company's experimental gene editors to see which of the molecular tools might safely correct the genetic defects, preventing future generations from suffering the same fate.
使用经过基因编辑的胚胎来生育婴儿是违反美国法律的。Tie希望向世界证明:利用她公司的先进基因编辑技术来对抗遗传性疾病所带来的好处,远远超过了其潜在风险。
Using gene-edited embryos to create babies is against U.S. law Tie wants to convince the world that the benefits of using her company's advanced gene editing technology to fight inherited diseases outweigh its risks.
长期以来,由于伦理和安全方面的顾虑,对人类胚胎进行基因编辑(这些基因编辑会遗传给后代)一直被视为禁忌行为;在美国,这种行为是非法的。她认为现在是时候重新审视这一禁令了。
Editing genes in human embryos that would be passed down for generations has long been taboo because of ethical and safety concerns. In the U.S., it's illegal. She says it may be time to reconsider.
“全球有数亿人患有各种遗传性疾病,比如亨廷顿舞蹈症、囊性纤维化、遗传性癌症以及泰-萨克斯病,”Tie说道。
"Hundreds of millions of people around the world have these diseases: Huntington's, cystic fibrosis, hereditary cancers, Tay-Sachs," says Tie.
“我们提出的方法具有革命性:在胚胎出生前,就利用这种精准的基因编辑技术修复其基因突变,从而让下一代不再携带这些疾病。这将会彻底改变我们所熟知的医学体系,”Tie解释道。
"What we are proposing is something completely revolutionary: apply this amazing precision genome editing to embryos that have a mutation before they are born," Tie says. "That allows the next generation to not be born with these diseases.
Origin公司还在利用人工智能的帮助,对其他携带致病突变的细胞进行基因编辑实验。这些细胞包括科学家在实验室中从成人皮肤或血液细胞培育出的诱导多能干细胞,以及另一种名为成纤维细胞的细胞。
It changes the entire paradigm of medicine as we know it." Origin is also testing gene editors designed with the help of artificial intelligence on other kinds of cells carrying disease-causing mutations. Those cells include induced pluripotent stem cells which scientists create in the lab from adult skin or blood cells, and other cells called fibroblasts.
Tie带领我们进入另一个房间,向我们展示了他们的研究成果——其中包括那些携带乳腺癌致病突变的细胞。她小心翼翼地从培养箱中取出一个透明塑料培养皿,将其放在显微镜下;显微镜将细胞图像投射到显示器上。
Tie leads us into another room to show us that work, including cells carrying a mutation for breast cancer that Origin is trying to repair. She gingerly lifts a clear plastic tray from an incubator and places it under a microscope that projects images of the cells onto a monitor.
“你们现在看到的是基因编辑工具正在进入细胞内部的过程,”她解释道。
"What you see here is the gene editor actually going into the cells," she says.
这些细胞带有增加患乳腺癌风险的基因突变,它们已经被注入了Origin公司开发的实验性基因编辑工具。在显微镜下,这些细胞呈现出荧光绿色;而那些颜色较浅的绿色区域则表明基因编辑工具已经成功进入了细胞内部。
Tie examines cells carrying a genetic mutation that increases the risk for breast cancer that have been injected with one of Origin's experimental gene editors. The cells look like fluorescent green blobs. Those glowing a lighter color green indicate the gene editor has successfully made it into the cell, she says.
“这意味着我们现在有了一个非常优秀的平台,可以将我们专有的基因编辑工具引入这些细胞,从而纠正突变,”泰说。“这只是第一步。”
"What this means is now we have a really great platform to introduce our proprietary gene editors into those cells to then correct the mutation," Tie says. "You're looking at the very first step."
对风险和伦理的担忧依然笼罩着这一领域。事实上,Origin目前仍处于起步阶段,美国至少还有另一家初创公司——位于旧金山的Preventive——也在从事这类工作。但考虑到创造基因编辑婴儿的前景所引发的重重复杂问题,以及该领域充满争议的历史,这些公司的存在本身就令人瞩目。
Concerns about risks and ethics hang over the field Indeed, it's still very early days for Origin, and at least one other U.S. startup is doing this sort of thing, Preventive in San Francisco. But the very existence of these companies is striking, given the thicket of issues raised by the prospect of creating gene-edited babies, and the controversial history of the field.
中国科学家贺建奎因在2018年宣布利用CRISPR基因编辑技术培育出首批转基因婴儿而震惊世界,随后被判处三年有期徒刑。
He Jiankui, a Chinese scientist, was imprisoned for three years after he stunned the world by announcing in 2018 that he had used CRISPR gene editing to create the first genetically modified babies.
当时,人们普遍认为这项技术还太新,无法确定它对婴儿或后代是否安全。基因编辑可能会无意中产生危险的突变,并代代相传。
At that time, the technology was considered far too new for anyone to know if it was safe for the babies — or future generations. The gene editing could have inadvertently created dangerous mutations that could then be passed down for generations.
大多数科学家认为,尝试利用基因修饰的人类胚胎来孕育婴儿仍然为时过早,而且可能永远无法保证安全。但是,诸如先导编辑和碱基编辑等更新、更精确的基因编辑技术,已经提高了该技术的准确性。一些科学家和生命伦理学家认为,这些进展带来了一线希望,即有朝一日出于预防某些毁灭性遗传病的目而使用基因编辑可能会变得符合伦理。
Most scientists think it's still premature to try to use genetically modified human embryos to produce babies — and it may never be safe. But newer, more precise gene-editing techniques, such as prime editing base editing have improved the accuracy of the technology Some scientists and bioethicists argue the advances raise hope they might one day be ethical to use gene editing to prevent some devastating genetic diseases.
然而,硅谷的风险投资家、东海岸的企业家和其他各方已经涌现,试图推动该领域向前发展。尽管如此,即便在那些认为经过修饰的人类胚胎有朝一日可能足够安全、能在极少数情况下用于预防罕见疾病的人当中,也有一些人对Origin Genomics公司感到担忧。
But already Silicon Valley venture capitalists, East Coast entrepreneurs and others have emerged to try to push the field forward Still, even among those people who think modified human embryos may some day be safe enough to be used in very limited circumstances to prevent rare diseases, some worry about Origin Genomics.
“这给了人们虚假的希望,”伦敦弗朗西斯·克里克研究所的生物学家罗宾·洛维尔-巴杰说。“我担心他们推进得太快,可能会犯下错误,从而拖累整个领域——人们或许曾寄希望于这些技术最终能帮到他们,但这种希望也将随之破灭。”基因编辑会被用于设计婴儿吗?
"It's giving people false hope," says Robin Lovell-Badge a biologist at the Francis Crick Institute in London. "And I am concerned that they are rushing ahead too fast and making some mistake, which would then set back the field – and the hope that people may have these techniques may eventually help them will be lost." Would gene editing be used for designer babies?
即使对人类胚胎进行编辑的安全性得到证实,一些人仍担心,一旦允许这种做法,就可能为设计婴儿和基因增强人类构成的反乌托邦未来打开大门。
Even if editing of human embryos could be proven to be safe, some fear allowing it at all could open the door to designer babies and a dystopian future of genetically enhanced people.
“我非常担心事态可能走向何方,”负责监督机构“遗传与社会中心”的凯蒂·哈森说。“这些公司背后的一些硅谷投资者非常坦率地阐述他们对未来的愿景。那是一个大规模生产基因工程人类的愿景,令人不寒而栗。”其他公司已经在筛查胚胎的智商、身高等特征,以及疾病风险。
"I'm very concerned about where this could be headed," says Katie Hasson who runs the Center for Genetics and Society, a watchdog group. "Some of the Silicon Valley investors behind these firms are very open about their vision for the future. It's a vision of mass-produced, genetically engineered humans that's horrifying." Other companies are already screening embryos for traits like IQ and height in addition to risk for diseases.
私营部门的工作让一些学术研究人员感到担忧。“我认为他们没有诚实地告诉我们他们真正这样做的动机,”加州大学伯克利分校研究基因编辑的费奥多尔·乌尔诺夫说。“他们实际上从事的是胚胎——进而最终是婴儿——改良的生意。”蒂表示,起源公司并非如此,她强调自己对帮助人们拥有健康孩子之外的任何事情都没有兴趣。
The work in the private sector worries some academic researchers. "I just don't think they're being honest with us about why they're really doing this," says Fyodor Urnov who studies gene editing at the University of California, Berkeley. "They're really in the business of embryo — and thus ultimately baby — improvement." Tie says that's not the case for Origin, stressing she has no interest in anything except helping people have healthy children.
一些人还担心,这些公司的出现可能导致海外涌现出缺乏监管的诊所,在监管较宽松的地区提供胚胎编辑服务。
Some also worry the advent of these companies may lead to unregulated clinics springing up overseas to offer embryo editing where regulations are looser.
“我非常担心基因编辑旅游现象,”威斯康星大学麦迪逊分校法学与生物伦理学荣誉退休教授R·阿尔塔·夏罗(R. Alta Charo)说道。她目前正为政府机构和私营公司担任顾问。“这是我对比接下来发展的最大担忧。”由于铁辉在臭名昭著的中国科学家出狱后曾与其有过个人接触,她的工作也因此受到了格外的审视。
"I'm very concerned about genome-editing tourism," says R. Alta Charo a professor emerita of law and bioethics at the University of Wisconsin-Madison who's now consulting for government agencies and private companies. "This is my fear for what comes next." Tie's work draws additional scrutiny because she was personally involved with the notorious Chinese scientist after he was released from prison.
但就铁辉而言,她表示这位不守规矩的科学家并未以任何方式参与Origin公司的业务。她承诺,自己的推进过程将非常缓慢且有条不紊,并配合独立的监督与透明度,以确保胚胎编辑的安全性。“这是一项威力巨大的技术。安全必须放在首位,”她说道。
But for her part, Tie says the rogue scientist is not involved in Origin in any way. She promises she's moving very slowly and methodically, with independent oversight and transparency to make sure embryo editing would be safe. "This is a very powerful technology. Safety must come first," she says.
铁辉希望Origin能够产生足够的证据,证明将基因修饰的人类胚胎用于生殖是安全的。
Tie hopes Origin will produce enough evidence to prove that it would be safe to use genetically modified human embryos for reproduction.
“我认为,如果你拥有一项已被证明安全的技术,并且它能帮助数以亿计饱受这些疾病折磨的人,那么不去进行这项研究才是不道德的,”铁辉说。“我的希望是,人类能够把握这项技术的积极面。”她希望Origin最终能产生足够的数据,来说服国会解除食品药品监督管理局(FDA)长期以来的禁令,不再禁止其审议涉及基因编辑人类胚胎的实验,同时也说服其他国家的监管机构。
"I think that if you have a technology that can be proven to be safe, and it can help hundreds of millions of people that suffer from these diseases, it is unethical to not pursue this research," Tie says. "My hope is that humanity can capture the upside of this technology." She hopes Origin will eventually produce enough data to convince Congress to lift a long-standing ban on the Food and Drug Administration even considering experiments involving gene-edited human embryos, as well as persuade regulators in other countries.
“临床医生、监管人员、生物伦理学家、患者维权人士——我们所有人都必须携手合作,确保这项技术以正确的方式被使用,”她说道。
"Clinicians, regulators, bioethicists, regulators, patient advocates – all of us must come together to make sure this technology is used in the right way," she says.
这是一个让儿童免受遗传性疾病之苦的机会。铁辉建议NPR(美国国家公共电台)与她正在合作的一个家庭见面。于是,一名记者和一名摄影师横跨全城,来到曼哈顿中城的一栋高层公寓楼,乘坐高速电梯直达顶层公寓,见到了妮可(Nicole)和阿萨夫·埃兹拉(Asaf Ezra)夫妇。
A chance to spare children from inherited diseases Tie suggests NPR meet with one of the families she's working with. So a reporter and photographer travel across town to visit a towering apartment building in midtown Manhattan and take the high-speed elevator to a penthouse to meet Nicole and Asaf Ezra.
现年38岁的妮可原籍英国。37岁的阿萨夫则来自以色列。他们很想生三个孩子。
Nicole, 38, is originally from the United Kingdom. Asaf, 37, is from Israel. They'd love to have three kids.
妮可(Nicole)和阿萨夫·埃兹拉(Asaf Ezra)一直在努力孕育不会遗传导致结直肠癌基因突变的孩子,这种基因突变世世代代困扰着妮可的家族。
Nicole and Asaf Ezra have been struggling to have children who won't inherit a genetic mutation that causes colorectal cancer and that has plagued Nicole's family for generations.
“我一直想要血脉相连的孩子。我想拥有自己的亲生骨肉,”妮可说。“我希望看到一个小小的我和他到处跑,然后觉得,‘没错,那就是我的孩子。’”但这对夫妇多年来一直在努力生孩子,因为妮可来自一个家族,世世代代都受到一种罕见但可怕的遗传病的困扰,这种病被称为家族性腺瘤性息肉病。基因突变会导致消化系统中形成被称为息肉的癌前病变。如果不切除,它们可能会癌变。
"I've always wanted physically my blood and flesh children. I want to have my biological children," Nicole says. "I want it to be a little me or him running around and be like, 'Yep, that's my kid.'" But the couple's been struggling to have children for years because Nicole comes from a family that's been plagued for generations by a rare but terrible genetic condition known as familial adenomatous polyposis A genetic mutation causes precancerous growths called polyps to form in the digestive system. They can become cancerous if they aren't removed.
“当我发现我们患有这种病时,我吓坏了,”妮可说。“这就像当头棒喝。非常艰难。”妮可13岁时不得不切除大部分大肠。她定期接受手术以发现并切除息肉。她生活在会患上结直肠癌的恐惧中,就像她的祖父、她的母亲和许多其他亲戚一样。“太可怕了,”她说。
"When I found out we had it, I was petrified," Nicole says. "It was a slam in the face. It was very hard." Nicole had to have most of her large intestine removed when she was 13. She regularly undergoes procedures to spot and remove polyps. She lives in fear she'll get colorectal cancer, like her grandfather, her mother, and so many other relatives. "It's horrible," she says.
因此,妮可经历了多轮试管婴儿(IVF)治疗,试图培育出未遗传该突变的胚胎。这对夫妇最终确实得到了一个健康的胚胎,但那次怀孕却不幸流产。这对夫妇仍在继续尝试。
So Nicole has gone through rounds of IVF to try to produce embryos that haven't inherited the mutation. The couple did finally get one healthy embryo, but lost that pregnancy to a miscarriage. The couple is still trying.
但阿萨夫也听说了起源基因组学公司(Origin Genomics)。
But Asaf also heard about Origin Genomics.
“我们只是不想把癌症传给我们的孩子,”阿萨夫说。“谁会愿意那样做呢,对吧?”因此,身为心理治疗师的妮可和在风险投资公司工作的埃兹拉投资了这家公司,并捐出了他们三个受影响的胚胎用于公司的研究。
"We just don't want to pass on cancer to our kids," Asaf says. "Who would want that, right?" So Nicole, a therapist, and Ezra, who works at a venture capital firm, invested in Origin, and donated three of their affected embryos for the company's research.
这对夫妇投资了起源基因组学公司,并捐出了他们的三个胚胎用于公司的研究,希望这能让他们生下没有导致结直肠癌基因突变的孩子。
The couple invested in Origin Genomics and donated three of their embryos for the company's research in the hopes that it might allow them to have children without the genetic mutation that causes colorectal cancer.
“这将帮助无数女性,效果令人惊叹,”妮科尔哽咽着说,强忍泪水。“我知道我现在在哭,因为我在思考这件事。但这是喜悦的泪水,因为人们将获得这样的机会。它将为世界带来巨大的好处,也有许多女性将不必像我一样经历创伤。”这对夫妇指出,当体外受精技术最初问世时,也曾引发过类似的担忧。他们认为,社会只需对这项技术进行规范,确保其被用于正当目的即可。
"The amount of women this would help would be amazing," says Nicole, choking back tears. "I know I'm crying now because I'm thinking about it. But it's really tears of joy for people with the chance that they'll get. It would do so much good for the world, and there's so many women who won't get the trauma that I've had." When it was first developed IVF raised similar fears, the couple notes. Society just needs to regulate this technology to make sure it's used for the right reasons, they say.
“我们不应因为害怕创造其他事物而放弃技术,”阿萨夫说。“我认为这并非道德上的制高点。”
"We shouldn't give up technology for fear of creating other things," Asaf says. "I don't think that's the moral high ground here."