Henninger's Research: Targeting RNA for Cancer Prevention and Treatment (2025)

Imagine a world where cancer isn't fought with harsh, one-size-fits-all treatments, but instead targeted with laser-like precision right at the genetic core of the disease— that's the groundbreaking vision Jonathan Henninger is helping to bring to life at Carnegie Mellon University. But here's where it gets really exciting: what if we could prevent cancer before it even starts, using the body's own molecular machinery? Stick around, because this isn't just science fiction; it's happening now, and it might just change everything we know about battling one of humanity's biggest killers.

Dated October 22, 2025, this article dives into Henninger's innovative work. For media inquiries, reach out to Cassia Crogan, who heads University Communications & Marketing, via email.

Henninger, an assistant professor in the Department of Biological Sciences at Carnegie Mellon, is at the forefront of a burgeoning field in life sciences. He's exploring how ribonucleic acid—commonly known as RNA—can be leveraged to create revolutionary treatments. RNA, for those just starting to dip their toes into biology, is a crucial molecule that acts like a messenger in our cells, helping to translate the instructions from DNA into proteins that keep our bodies functioning. But as Henninger and his colleagues are discovering, RNA does far more than just deliver messages; it plays a pivotal role in shaping how genes are expressed and how cells are organized.

Delving deeper into RNA's role in gene expression and cellular organization, Henninger's research focuses on gene expression—the process where the DNA blueprint is 'read' by the cell to produce proteins. To start, RNA copies are made from DNA, and these serve as templates for making specific proteins. Yet, RNA's influence extends beyond this basic function. It actively shapes multiple stages of gene expression, including by regulating tiny, membrane-less compartments inside cells. These structures, formed by proteins and nucleic acids, are called biomolecular condensates, and they function like specialized hubs where molecules cluster to perform coordinated biological tasks.

As Henninger explains, 'RNA acts as a very important scaffold that these condensates form around.' He likens proteins to chess pieces, each with unique moves and roles, while DNA and RNA are the chessboard itself—determining how the pieces arrange and interact. This analogy highlights the deep connection between RNA's structure and properties and how they influence the creation and breakdown of condensates tied to gene expression. And this is the part most people miss: these condensates aren't just passive; they're dynamic workstations that can go awry, leading to diseases like cancer.

Shifting to precision medicine at the genetic level through RNA-based therapies, many illnesses, particularly cancer, stem from errors in gene expression. Traditionally, drugs haven't been great at zeroing in on these processes to fix them and restore cell health. The genomic revolution has uncovered thousands of genetic variations associated with diseases, many in areas that control gene activity. Henninger suspects some of these variations interfere with RNA's function—and that's exactly what his lab is probing.

His team is crafting cutting-edge tools to examine RNA and visualize transcription, the initial phase where genes are switched on in cells. A key area of interest is how mutations in RNA-binding proteins fuel blood cancers, such as acute myeloid leukemia. 'Mutations in RNA-binding proteins or changes in RNA levels disrupt condensates and gene expression, paving the way for cancer to develop,' Henninger notes. 'We're expanding this research to real patient cancer cells, teaming up with clinicians at UPMC.' Plus, collaborations with CMU experts like Dr. Andreas Pfenning and Dr. Martin Zhang, who specialize in machine learning and genomics, are enhancing their work.

Another avenue Henninger's lab is pursuing involves antisense oligonucleotides, or ASOs—man-made, single-stranded RNA molecules engineered to latch onto specific messenger RNA sequences. These can rectify gene expression glitches, addressing disorders from the root. Teaming up with Subha Das, an associate professor of chemistry, and Bruce Armitage, the head of chemistry and co-director of the Center for Nucleic Acid Science and Technology at CMU, they're advancing these efforts. 'We're eager to automate testing of various ASOs and employ AI to uncover design principles,' Henninger shares. This automation could speed up discoveries, making RNA therapies more accessible and efficient.

Backing from generous foundations like the Shurl and Kay Curci, Charles E. Kaufman, and Samuel and Emma Winters is propelling Henninger's lab forward. This support funds crucial experiments, innovative RNA tests, and advanced imaging of gene expression. 'Our lab's initial breakthroughs and promising results wouldn't have been feasible without the backing of foundations passionate about basic science—we're deeply thankful,' he says.

Highlighting collaborative science in Pittsburgh, Henninger relocated to the area in early 2024, having grown up just an hour away in Indiana, Pennsylvania, to join Carnegie Mellon. 'Pittsburgh boasts a rare blend of chemistry, physics, AI, and biology all converging on shared objectives. It's incredibly influential,' he remarks. 'I'm right in the sweet spot, surrounded by specialists for every aspect of our research.' This teamwork shone brightly at a recent CMU conference, where over 100 experts from biology, chemistry, physics, engineering, and medicine convened to discuss condensates.

Beyond research, Henninger is dedicated to training the next generation of researchers. In the last 18 months, he's assembled a vibrant team of about six graduate students. 'I'm a big believer in collaborative science, as it allows parallel efforts toward common goals, accelerating discoveries and making the process enjoyable,' he explains. He's also impressed by the undergraduates at CMU who've joined his lab. 'They're performing at graduate-level standards,' he says. 'Their brilliance, fresh ideas, and enthusiasm make working with them a thrill.'

But here's where it gets controversial: While RNA-targeted therapies hold immense promise for precision cancer treatment, critics might argue that tinkering with gene expression could have unforeseen side effects, like off-target impacts on healthy cells or even ethical dilemmas around 'editing' human biology. Is this the dawn of a cure-all era, or are we risking a Pandora's box of genetic manipulations? And this is the part that sparks debate: Some say traditional chemotherapy and radiation, though blunt, have proven track records—why switch to something experimental that might not work for everyone? What do you think—will Henninger's RNA innovations truly transform cancer care, or should we be cautious about rushing into these molecular frontiers? Share your thoughts in the comments below; do you agree that precision RNA therapies are the future, or disagree that the risks outweigh the benefits? Your opinions could fuel the conversation!

Henninger's Research: Targeting RNA for Cancer Prevention and Treatment (2025)
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