【中美创新时报2024 年 10 月 8 日编译讯】（记者温友平编译）周一，两位马萨诸塞州的研究科学家因发现微小的构建块（称为 microRNA）而获得诺贝尔医学奖，这种构建块控制着植物和动物的细胞活动，并帮助催生了马萨诸塞州及其他地区越来越多的药物发现活动。《波士顿环球报》记者罗伯特·魏斯曼、艾米丽·斯威尼和伊兹·布莱尔斯对此作了下述报道。
该奖项是科学界最负盛名的奖项，由哈佛医学院遗传学教授、波士顿麻省总医院成员加里·鲁夫昆 (Gary Ruvkun)博士和伍斯特麻省大学陈曾熙医学院自然科学教授维克多·安布罗斯 (Victor Ambros)博士共同获得。
他们的研究成果被 Alnylam Pharmaceuticals 等先锋公司用于抑制导致癌症、心脏病和其他疾病的基因。
在周一上午的新闻发布会上，Ruvkun 赞扬了麻省总医院实验室的研究人员及其合作者创造了一个可以实现科学突破的工作环境。“研究遗传学的乐趣在于惊喜，”Ruvkun 说。“大约有 20,000 个基因，我知道其中的 500 个，也许 1,000 个。”
核糖核酸，或 RNA，是一种携带细胞如何产生蛋白质指令的分子。 MicroRNA 是这个过程的控制者，如果产生的蛋白质过多或过少，这个过程就会出错并导致疾病。Ruvkun 和 Ambros 于 1993 年在蛔虫身上进行了初步研究，但他们的发现适用于所有多细胞生物。
麻省总医院分子生物学系主任 Jeannie Lee 博士表示，当时人们并没有广泛了解 microRNA 的重要性，部分原因是最初的研究是在微小的蠕虫身上进行的——Ruvkun 称其为“植物上最厉害的生物”。
“microRNA 的神奇之处在于，它们不只是一次针对一个东西，它们实际上针对的是整个基因网络，”Lee 说。“这是一种对基因表达有巨大影响的微小 RNA。”
瑞典诺贝尔委员会在周一的一份声明中表彰了这些科学家，因为他们帮助研究界理解了控制基因活动的关键原理。委员会秘书长托马斯·珀尔曼 (Thomas Perlmann) 表示，他们的发现“对生物体的发育和功能至关重要”。
周一，这两位头发凌乱的科学家分别在波士顿和伍斯特的新闻发布会上发表了讲话——鲁夫昆身穿绿色 Polo 衫，安布罗斯身穿鲜艳的夏威夷印花衫。
安布罗斯是新罕布什尔州汉诺威人，毕业于麻省理工学院，在麻省大学医学院任教 15 年。他说，由于手机关机，他错过了诺贝尔委员会的清晨电话。
最终有人联系到了他住在康涅狄格州的成年儿子格雷格，无法联系到父亲，他打电话给安布罗斯的妻子罗莎琳德“坎迪”李，她是麻省大学的科学家和研究合作者。安布罗斯说，当她传达这个消息和儿子的留言时，他感到“很惊讶”，儿子说“瑞典人打来电话时请接电话”。
“我希望这一发现的认可将进一步激发研究 RNA 的年轻科学家的热情，”安布罗斯在周一的采访中说道，“因为 RNA 是我们细胞中令人惊奇的组成部分。也许它将激发更多人进入该领域。”
鲁夫昆周一告诉记者，他不习惯新闻发布会、电视摄像机和诺贝尔奖带来的高度关注。“这是一个完全不同的世界，”他说。但他说，几年前他曾陪同一位获得诺贝尔奖的同事去过斯德哥尔摩，并回忆说，“他们知道如何聚会。”
鲁夫昆出生于加州伯克利，在哈佛大学获得博士学位，在麻省理工学院担任博士后研究员，并于 1985 年成为麻省总医院和哈佛医学院的首席研究员。
安布罗斯和鲁夫昆都曾在麻省理工学院 H. Robert Horvitz 的实验室担任博士后，后者于 2002 年获得诺贝尔奖。霍维茨是麻省理工学院的生物学教授，也是霍华德休斯医学研究所的研究员，他说安布罗斯和鲁夫昆早就该获得诺贝尔奖了。
“诺贝尔奖并不总是立竿见影的，”霍维茨周一表示。“但我可以说，当我 2002 年去斯德哥尔摩时，我谈到的一件事就是他们的工作具有开创性。所以到 2002 年，在我看来，他们应该获得这个奖项是完全清楚的。”
Horvitz 表示，Ambros 和 Ruvkun“做出了真正开创性的贡献，而且贡献方式非常互补”。 “他们的整个合作努力可以说是合作的典范。除此之外，他们两人都是杰出的科学家。”
他们的工作强调了马萨诸塞州公共和私人研究机构在理解遗传学方面的主导作用。
马萨诸塞大学校长 Marty Meehan 称波士顿-伍斯特合作是该州在生物医学创新方面全球影响力的象征。“这是该州世界级机构合作的典范，”他说。
周一，马萨诸塞大学陈医学院校长 Michael F. Collins 提到了医学院扎根的“世界领先”RNA 研究社区。该学院分子医学教授 Craig Mello 博士与加州斯坦福医学院的 Andrew Z. Fire 因发现 RNA 干扰而荣获 2006 年诺贝尔医学奖。
梅洛周一表示，马萨诸塞大学是在他的敦促下招募了安布罗斯，如果没有安布罗斯，他自己在 2006 年获得诺贝尔奖也是不可能的。梅洛也曾与鲁夫昆共事，他说这两位新诺贝尔奖得主“都是了不起的人，充满好奇心和对科学的热情。”
麻省总医院官员表示，对 microRNA 在诊断、预后和治疗多种遗传疾病方面的潜力的研究已经从 30 多年前鲁夫昆和安布罗斯发表的两篇原始论文扩展到今天的 176,000 篇论文。
科学家现在认为，大多数植物和动物基因组（包括人类基因组）都含有 1,000 多个 microRNA，它们控制与细胞活动和疾病有关的蛋白质编码核酸。基于 microRNA 的实验性治疗目前正在进行心脏病、癌症和神经退行性疾病的临床试验。
最近的诺贝尔奖也凸显了所谓的“信使 RNA”在理解疾病遗传原因方面的核心作用。去年，诺贝尔生理学或医学奖颁给了宾夕法尼亚大学的匈牙利裔美国人 Katalin Karikó 和美国人 Drew Weissman，以表彰他们发现的 mRNA 疫苗的研制成功，这些疫苗对减缓 COVID 大流行至关重要。
自 1901 年以来，诺贝尔医学奖已颁发 114 次，共有 227 位获奖者。该奖项的现金奖励为 1100 万瑞典克朗，相当于约 100 万美元，来自奖项创始人、瑞典发明家阿尔弗雷德·诺贝尔的遗赠。
获奖者将受邀于 12 月 10 日（诺贝尔逝世周年纪念日）在斯德哥尔摩举行的颁奖典礼上领奖。
诺贝尔奖的公告仍在继续，物理学奖将于周二公布，化学奖将于周三公布，文学奖将于周四公布。诺贝尔和平奖将于周五公布，经济学奖将于 10 月 14 日公布。
本报告使用美联社的材料。《环球报》员工 Travis Andersen 对此亦有贡献。
题图：分子生物学家加里·鲁夫昆 (Gary Ruvkun) 与维克多·安布罗斯 (Victor Ambros) 共同获得 2024 年诺贝尔生理学或医学奖后，在麻省总医院举行的新闻发布会上发表讲话。David L. Ryan/Globe Staff
附原英文报道：
Two Massachusetts professors awarded Nobel Prize in medicine
Research scientists at MGH, UMass honored for their discovery of microRNA
By Robert Weisman, Emily Sweeney and Izzy Bryars Globe Staff and Globe Correspondent,Updated October 7, 2024
Molecular biologist Gary Ruvkun speaks during a press conference after winning a shared 2024 Nobel Prize with Victor Ambros in Physiology or Medicine at Massachusetts General HospitalDavid L. Ryan/Globe Staff
Two Massachusetts research scientists were awarded the Nobel Prize in Medicine on Monday for their discovery of tiny building blocks, called microRNA, that control the cellular activity of plants and animals and have helped spawn a growing cluster of drug discovery activity in Massachusetts and beyond.
The award — science’s most prestigious — will be shared by Dr. Gary Ruvkun, a professor of genetics at Harvard Medical School and a member of the Mass General Research Institute in Boston, and Dr. Victor Ambros, a professor of natural science at the University of Massachusetts Chan Medical School in Worcester.
Their findings are being used at pioneering companies such as Alnylam Pharmaceuticals to deactivate genes that contribute to cancers, heart disease, and other disorders.
At a news conference Monday morning, Ruvkun credited the researchers in his MGH lab and their collaborators for creating a work environment where scientific breakthroughs can occur. “The joy of doing genetics … is the surprises,” Ruvkun said. “There’s 20,000 genes, approximately, and I know something about 500, maybe 1,000 of them.”
Ribonucleic acid, or RNA, is a molecule that carries instructions to cells on how to produce proteins. MicroRNA acts as the controller of this process, which can go awry and result in diseases if too much or too little protein is created. Ruvkun and Ambros did their initial research in 1993 in roundworms, but their findings apply to all multicellular organisms.
Dr. Jeannie Lee, who chairs the molecular biology department at Mass General, said the importance of microRNAs was not widely understood at the time partly because initial research was done on the tiny worms — which Ruvkun called “the most bad-ass organisms on the plant.”
“The amazing thing about microRNAs is that they’re not just targeting one thing at a time, they’re actually targeting a whole network of genes,” Lee said. “It’s a tiny little RNA with a huge impact on gene expression.”
In a statement Monday, the Nobel Committee in Sweden cited the scientists for helping the research community understand a key principle governing how gene activity is regulated. Their discovery is “proving to be fundamentally important for how organisms develop and function,” said Thomas Perlmann, secretary-general of the committee.
The tousled-haired scientists spoke at separate press conferences in Boston and Worcester on Monday — Ruvkun wearing a green Polo shirt, Ambros a bright Hawaiian print.
Ambros, a native of Hanover, N.H., and an MIT graduate who has been on the UMass medical school faculty for 15 years, said he missed the Nobel Committee’s early morning call because his phone was off.
Someone ultimately reached his adult son Greg, in Connecticut, and unable to reach his father, he called Ambros’s wife, Rosalind “Candy” Lee, a UMass scientist and research collaborator. Ambros said he was “astonished” when she conveyed the news and a message from their son to “pick up the phone when someone from Sweden is calling.”
“I’m hoping that the recognition of this discovery will further stimulate excitement among young scientists who study RNA,” Ambros said in an interview Monday, “because RNA is an amazing constituent of our cells. Perhaps it will stimulate more people to come into the field.”
Ruvkun told reporters Monday that he wasn’t accustomed to news conferences, TV cameras, and the heightened attention that comes with the Nobel Prize. “It’s a completely different world,” he said. But he said he’d accompanied a colleague who’d won a Nobel to Stockholm some years ago and recalled, “They know how to party.”
Ruvkun, a native of Berkeley, Calif., received his PhD from Harvard University, was a postdoctoral fellow at Massachusetts Institute of Technology, and became a principal investigator at Massachusetts General Hospital and Harvard Medical School in 1985.
Ambros and Ruvkun both worked as postdocs in the MIT lab of H. Robert Horvitz, who won the Nobel Prize in 2002. Horvitz, a biology professor at MIT and an investigator at the Howard Hughes Medical Institute, said the Nobel Prize was long overdue for Ambros and Ruvkun.
”The Nobel isn’t always instantaneous,” Horvitz said Monday. “But I can say that when I went to Stockholm in 2002, one of the things I talked about was their work as being fundamentally groundbreaking. So by 2002 in my mind, it was perfectly clear they ought to get this prize.”
Ambros and Ruvkun “made truly pioneering contributions and in very complementary ways,” Horvitz said. “Their whole collaborative effort was a sort of poster child for what a collaboration can be. And then, coupled with all of this, both of them are brilliant scientists.”
Their work underscores the leading role of public and private Massachusetts research institutions in understanding genetics.
University of Massachusetts president Marty Meehan cited the Boston-Worcester collaboration as emblematic of the state’s global footprint in biomedical innovation. “It is an example of what happens when the state’s world-class institutions work together,” he said.
On Monday, UMass Chan Medical School chancellor Michael F. Collins cited the “world-leading” RNA research community that has taken root at the medical school. Dr. Craig Mello, a professor of molecular medicine at the school, was awarded the 2006 Nobel Prize for Medicine, along with Andrew Z. Fire of the Stanford School of Medicine in California, for the discovery of RNA interference.
Mello said Monday that UMass recruited Ambros at his urging and his own 2006 Nobel Prize wouldn’t have been possible without Ambros. Mello, who also worked with Ruvkun, said the two new Nobel laureates “are incredible human beings, full of curiosity and a passion for science.”
MGH officials said research into the potential of microRNAs for the diagnosis, prognosis, and treatment of a wide range of genetic diseases has expanded from the two original papers published by Ruvkun and Ambros more than three decades ago to 176,000 papers today.
Scientists now believe that most plant and animal genomes, including the human genome, contain more than 1,000 microRNAs, which control protein-coding nucleic acids involved in cellular activity and diseases. Experimental treatments based on microRNAs are currently in clinical trials for heart disease, cancer, and neurodegenerative diseases.
Recent Nobel Prizes have also highlighted the centrality of so-called “messenger RNA” in understanding genetic causes of diseases. Last year, the Nobel Prize in Physiology or Medicine went to Hungarian-American Katalin Karikó and American Drew Weissman at the University of Pennsylvania for discoveries that enabled the creation of mRNA vaccines that were critical in slowing the COVID pandemic.
The Nobel Prize in Medicine has been awarded 114 times to a total of 227 laureates since 1901. The prize carries a cash award of 11 million Swedish kronor, equal to about $1 million, from a bequest left by the prize’s creator, Swedish inventor Alfred Nobel.
The laureates are invited to receive their awards at ceremonies in Stockholm scheduled for Dec. 10, the anniversary of Nobel’s death.
Nobel announcements continue with the physics prize on Tuesday, chemistry on Wednesday, and literature on Thursday. The Nobel Peace Prize will be announced Friday and the economics award Oct. 14.
Material from the Associated Press was used in this report. Travis Andersen of the Globe staff contributed.
