RNA therapeutics Archives | ��ϳԹ��Դ

Short-circuiting pancreatic cancer

Nick Wurm — Thu, 08 Jan 2026 16:00:45 +0000

Pancreatic ductal adenocarcinoma (PDAC) is the most lethal form of pancreas cancer. It’s also the most common form of the disease. Potential treatments typically target a key mutated oncogene called KRAS. In some cases, PDAC tumors with these mutations have resisted therapeutic efforts. However, combination therapies involving alternative drug targets may one day help clinicians...

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Breast cancer case study could inform clinical trials

Jen A. Miller — Mon, 10 Nov 2025 12:30:14 +0000

MALAT1 (pink) than the surrounding tissues." data-description="Fluorescent microscopy image showing clusters of blue and purple cells on a black background." />When researchers look for potential cancer therapy targets, they typically go after protein-coding genes that participate in disease progression. Disrupt them and negate whatever role they play in cancer’s development or spread. Researchers at ��ϳԹ��Դ (CSHL) have also been working on another kind of target. They’re studying a long non-coding RNA called...

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Special edition: Breast Cancer Awareness Month

Communications Department — Thu, 23 Oct 2025 11:30:49 +0000

Today, the five-year survival rate for early-stage breast cancer is about 99%. Awareness drives detection. But what about those breast cancers that aren’t caught early? What about those that evade common therapies? For Breast Cancer Awareness Month, we sat down with CSHL Professor David Spector to discuss some of his lab’s latest research and the...

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“Philifant” sculpture honors research leading to Spinraza

Nick Wurm — Fri, 29 Aug 2025 14:40:15 +0000

On August 27, pioneering digital artist Phil L. Herold donated a chrome “Philifant” sculpture to ��ϳԹ��Դ (CSHL) for the Krainer lab’s transformative, life-saving research on spinal muscular atrophy (SMA). CSHL Professor Adrian Krainer’s work led to the development of Spinraza, the first FDA-approved treatment for SMA. At the time, the neurodegenerative disease...

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Can you ace this SMA quiz?

Communications Department — Thu, 07 Aug 2025 11:30:37 +0000

{“questions”:{“8r78v”:{“id”:”8r78v”,”mediaType”:”image”,”answerType”:”text”,”imageCredit”:””,”image”:”https:\/\/www.cshl.edu\/wp-content\/uploads\/2025\/07\/Richard_Roberts_Phillip_Sharp.jpg”,”imageId”:”74800″,”video”:””,”imagePlaceholder”:””,”imagePlaceholderId”:””,”title”:”In 1993, Richard Roberts and Phillip Sharp won the Nobel Prize in Physiology or Medicine for which revolutionary discovery?”,”desc”:”While CSHL played a role in all of these groundbreaking discoveries, only one is attributable to Roberts and Sharp. In 1977, CSHL\u2019s Richard Roberts and MIT\u2019s Phillip Sharp co-discovered split genes and RNA splicing while working with...

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��ϳԹ��Դ hope to thousands of families

Jen A. Miller — Tue, 05 Aug 2025 11:30:41 +0000

It’s a parent’s worst nightmare, finding out your newborn child may never walk or even stand on their own. That used to be the reality for as many as one in 6,000 babies born with spinal muscular atrophy (SMA), a genetic disorder that causes extreme nerve damage, muscle weakness, and sometimes, paralysis. For a long...

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Adrian Krainer wins 2025 Heinrich Wieland Prize

Nick Wurm — Wed, 16 Jul 2025 12:30:07 +0000

��ϳԹ��Դ Professor Adrian Krainer has won the 2025 Heinrich Wieland Prize. Awarded annually by the Boehringer Ingelheim Foundation, the €250,000 prize recognizes exceptional scientists whose pioneering research pushes the boundaries of knowledge in biochemistry, biology, chemistry, and physiology. Krainer received the award for his revolutionary RNA splicing research, which led to the...

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A new “link” to triple-negative breast cancer

Margaret Osborne — Mon, 30 Jun 2025 14:00:38 +0000

LINC01235 is seen in red." data-description="" />Breast cancer is one of the most common cancers among women in the United States. Thanks to decades of fundamental research, it’s also one of the most curable. The exception is a particularly aggressive variant known as triple-negative breast cancer (TNBC). It accounts for 10 to 15 percent of all breast cancer cases. It disproportionately...

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Nature is trying to tell you something

Jen A. Miller — Mon, 03 Feb 2025 12:30:52 +0000

“Nature is trying to tell you something.” Taken by itself, this quote from Nobel laureate and former ��ϳԹ��Դ (CSHL) scientist Richard Roberts suggests that the solutions to any number of problems may be within earshot. If only things were so simple. The fact is, scientific experiments go awry all the time. When...

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Big clues emerge in small RNA mystery

Jen A. Miller — Mon, 16 Dec 2024 12:30:00 +0000

Arabidopsis plant (as seen above) is loaded with a molecule called pseudouridine." data-description="" />AI generated voice with Rob Martienssen’s actual voice Not everything inside us is, strictly speaking, us. The closer we look at the genome, the more we appreciate the role of small RNAs in what we call epigenetic inheritance. That’s when traits get passed down without altering our basic DNA sequence. We now know that small...

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Lifesaving life science with Katalin Karikó

Lisa Cruz — Thu, 12 Dec 2024 12:30:06 +0000

City of Science event presented in partnership between the CUNY Graduate Center and ��ϳԹ��Դ Center for Humanities & History of Modern Biology with support from the BGI Nobel Laureates Archives Program. Image: Wild Sound Photography" data-description="" />She helped save millions of lives. So, how does it feel? With humility and wit, 2023 Nobel laureate Katalin Karikó shared insights on her life’s work during a free public event at the CUNY Graduate Center on November 15. CUNY’s recital hall was filled to capacity as guests took their seats for an up-close and...

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2024 Double Helix Medal recipient Dr. Katalin Karikó

Communications Department — Tue, 10 Dec 2024 12:30:20 +0000

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19th annual Double Helix Medals dinner raises $7 million

Communications Department — Fri, 15 Nov 2024 21:00:55 +0000

On November 14, ��ϳԹ��Դ (CSHL) held its 19th annual Double Helix Medals dinner (DHMD) at the American Museum of Natural History in New York City. The event, emceed by CBS journalist Lesley Stahl, honored Alisa and Daniel Doctoroff and 2023 Nobel laureate Dr. Katalin Karikó. With the support of the event chairs...

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Why some RNA drugs work better than others

Jennifer Michalowski — Wed, 06 Mar 2024 12:30:20 +0000

Spinal muscular atrophy, or SMA, is the leading genetic cause of infant death. Less than a decade ago, ��ϳԹ��Դ (CSHL) Professor Adrian Krainer showed this brutal disease can be treated by tweaking a process called RNA splicing. This breakthrough resulted in Spinraza, the first effective treatment for SMA. It also opened a...

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Adrian Krainer awarded honorary IADR membership

Nick Wurm — Wed, 21 Jun 2023 23:00:43 +0000

��ϳԹ��Դ Professor Adrian Krainer has been named an honorary member of the International Association for Dental Research (IADR) for 2023. The IADR honored Krainer for fundamental and translational research that led to a life-saving treatment for spinal muscular atrophy (SMA). The group also acknowledged him for “enhancing our understanding of how [RNA]...

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Krainer named Society for RNA Therapeutics board member

Samuel Diamond — Tue, 18 Apr 2023 11:30:59 +0000

If RNA therapeutics were once considered the future of medicine, that future is now. Studies suggest RNA-based treatments have saved tens of millions of lives over the past few years. Now, the newly formed Society for RNA Therapeutics has come together to help further this cutting-edge science. On April 10, ��ϳԹ��Դ Professor...

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From tragedy, a potential pediatric cancer treatment

Nick Wurm — Wed, 12 Apr 2023 18:00:35 +0000

Diffuse intrinsic pontine glioma (DIPG) is a lethal pediatric brain cancer that often kills within a year of diagnosis. Surgery is almost impossible because of the tumors’ location. Chemotherapy has debilitating side effects. New treatment options are desperately needed. ��ϳԹ��Դ Professor Adrian Krainer is best known for his groundbreaking research on antisense...

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A molecular machine’s secret weapon exposed

Luis Sandoval — Thu, 23 Feb 2023 16:00:39 +0000

RNAs are having a moment. The foundation of COVID-19 vaccines, they’ve made their way from biochemistry textbooks into popular magazines and everyday discussions. Entire companies have launched dedicated to RNA research. These tiny molecules are traditionally known for helping cells make proteins, but they can do much more. They come in many shapes and sizes,...

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A new treatment approach for cystic fibrosis

Luis Sandoval — Thu, 14 Jul 2022 20:00:17 +0000

Antisense oligonucleotides, or ASOs, are molecules that can be used to control protein levels in cells. ��ϳԹ��Դ Professor Adrian Krainer leveraged ASO technology to develop the first FDA-approved treatment for spinal muscular atrophy called Spinraza®. The drug has helped over 11,000 patients make more of a protein that certain neurons in the...

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The promising drug duo that may improve SMA treatment

Luis Sandoval — Mon, 11 Jul 2022 11:30:15 +0000

In 2016, Spinraza® became a game-changer for spinal muscular atrophy (SMA) patients. It was the first FDA-approved treatment for the neurodegenerative disease, which is the leading genetic cause of infant death. The drug was conceived and developed by ��ϳԹ��Դ (CSHL) Professor Adrian Krainer and collaborators. But Krainer didn’t stop there. His lab...

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RNA therapeutics Archives | ���ϳԹ���Դ