Breakthrough in RNA Research Could Lead to Treatment for Neuromuscular Disorders

Danith Ly said this discovery paves the way for developing highly selective, structure-based RNA therapies with fewer side effects and broader applications.
Photo Credit: Courtesy of Carnegie Mellon University

Scientific Frontline: Extended "At a Glance" Summary

The Core Concept: Researchers have developed precise synthetic molecules, likened to "pothole fillers," that neutralize the toxic RNA repeats responsible for genetic neuromuscular disorders like myotonic dystrophy type 1 (DM1).

Key Distinction/Mechanism: Unlike traditional antisense therapies that require unwinding complex RNA structures to work, these ligands utilize "Janus" (bifacial) bases that insert themselves directly between RNA strands. This allows the molecule to bind to both sides of the toxic "hairpin" structure simultaneously, displacing harmful proteins without disturbing healthy RNA functions.

Origin/History: Published on January 15, 2026, by a team led by Professor Danith Ly at Carnegie Mellon University, this breakthrough builds upon years of research into peptide nucleic acids (PNAs) supported by the DSF Charitable Foundation since 2014.

Chemists determine the structure of the fuzzy coat that surrounds Tau proteins

MIT chemists showed they can use nuclear magnetic resonance (NMR) to decipher the structure of the fuzzy coat that surrounds Tau proteins. The findings may aid efforts to develop drugs that interfere with Tau buildup in the brain.
Image Credit: Jose-Luis Olivares, MIT; figure courtesy of the researchers
(CC BY-NC-ND 4.0)

Scientific Frontline: "At a Glance" Summary

• Discovery: MIT chemists successfully determined the atomic-level structure of the intrinsically disordered "fuzzy coat" surrounding Tau protein fibrils, a region comprising approximately 80% of the protein that was previously uncharacterizable by standard imaging.
• Methodology: The team developed a novel nuclear magnetic resonance (NMR) technique to magnetize protons within the rigid protein core and measure the transfer time to mobile segments, allowing them to map the proximity and dynamic movement of the disordered layers.
• Structural Detail: The analysis revealed a "burrito-like" architecture where the fuzzy coat wraps in layers around a rigid beta-sheet inner core, rather than extending randomly into the surrounding environment.
• Mechanism: The coat exhibits three distinct zones of mobility: a rigid core, an intermediate layer, and a highly dynamic outer layer rich in positively charged proline residues that are electrostatically repelled by the positively charged core.
• Significance: This structural model suggests that normal Tau proteins likely accumulate at the ends of existing filaments to drive fibril growth, rather than piling onto the sides, offering a precise mechanism for how Alzheimer's tangles propagate.
• Implication: Future therapeutic strategies must account for this protective layering, as small-molecule drugs intended to disaggregate Tau fibrils will need to effectively penetrate the dense fuzzy coat to reach and disrupt the toxic core.

New study shows how the cell repairs its recycling stations

Leaks in the cell's lysosomes can be life-threatening. The discovery by researchers Yaowen Wu and Dale Corkery may help to understand and prevent diseases such as Alzheimer’s.
Photo Credit: Yue Li

When the cell’s recycling stations, the lysosomes, start leaking, it can become dangerous. Toxic waste risks spreading and damaging the cell. Now, researchers at Umeå University have revealed the molecular sensors that detect tiny holes in lysosomal membranes so they can be quickly repaired – a process crucial for preventing inflammation, cell death, and diseases such as Alzheimer’s. 

Lysosomes are the cell’s recycling stations, handling cellular waste and converting it into building blocks that can be reused. Lysosomal membranes are frequently exposed to stress from pathogens, proteins, and metabolic byproducts. Damage can lead to leakage of toxic contents into the cytoplasm, which in turn may cause inflammation and cell death. Until now, the mechanism by which cells detect these membrane injuries has remained unknown. 

The Mechanical Ratchet: A New Mechanism of Cell Division Uncovered

A zebrafish embryo during the first cell division cycle, with the structural protein actin labelled, which marks the cell boundary and ingressing furrow. The image shows a time course from dark orange (before ingression) to brighter orange and finally white as ingression proceeds.
Image Credit: © Alison Kickuth, Brugués Lab

Cell division is an essential process for all life on earth, yet the exact mechanisms by which cells divide during early embryonic development have remained elusive – particularly for egg-laying species. Scientists from the Brugués group at the Cluster of Excellence Physics of Life (PoL) at Dresden University of Technology have revealed a novel mechanism that explains how early embryonic cells may divide without forming a complete contractile ring, traditionally seen as essential for this process. The findings, published in Nature, challenge the long-standing textbook view of cell division, revealing how parts of the cytoskeleton, and material properties of the cell interior (or cytoplasm) cooperate to drive division through a ‘ratchet’ mechanism.     

Exposure to natural light improves metabolic health

The research team provides the first evidence of the beneficial impact of natural light on people with this condition.
Image Credit: © Loïc Metz, UNIGE AI generated

Metabolic diseases have reached epidemic proportions in our society, driven by a sedentary lifestyle coupled with circadian misalignment - a desynchrony between our intrinsic biological clocks and environmental signals. Furthermore, we spend almost 90% of our time indoors, with very limited exposure to natural daylight. To investigate the specific role of daylight in human metabolism, particularly in glycemic control, researchers from the University of Geneva (UNIGE), the University Hospitals of Geneva (HUG), Maastricht University, and the German Diabetes Center (DDZ) conducted a controlled study with thirteen volunteers with type 2 diabetes. When exposed to natural light, participants exhibited more stable blood glucose levels and an overall improvement in their metabolic profile. These results, published in the journal Cell Metabolism, provide the first evidence of the beneficial impact of natural light on people with type 2 diabetes. 

Capturing the moment a cell shuts the door on free radicals

The moment a cell shuts the door on free radicals.
Illustration Credit: Catrin Jakobsson, Lund University

For the first time, researchers have been able to show how a cell closes the door to free radicals – small oxygen molecules that are sometimes needed, but that can also damage our cells. The study is published in Nature Communications and was led by Lund University. 

For our cells to function, they need to maintain a careful balance between beneficial and harmful oxygen molecules known as free radicals. One of the most important is hydrogen peroxide – the same substance found in disinfectants, but which our cells use in very small amounts to send important signals. However, in excessive concentrations, hydrogen peroxide can cause damage and even cell death.  

Researchers identify kidney sensor that helps control fluid balance

Rose Hill, Ph.D., second from left,studies sensory nerves within the kidneys at OHSU. Her new study identified a protein that acts as a pressure sensor in the kidneys, which helps the body control fluids and blood pressure. With her are lab team members: Taylor Krilanovich, Lily Schainker and Janelle Doyle.
 Photo Credit: OHSU/Christine Torres Hicks

A new study has identified a critical “pressure sensor” inside the kidney that helps the body control blood pressure and fluid levels. The finding helps explain how the kidneys sense changes in blood volume — something scientists for decades have known occurs but didn’t have a mechanistic explanation.

Researchers at Oregon Health & Science University and collaborating institutions discovered that a protein called PIEZO2 acts as a mechanical sensor in the kidney. When blood volume changes, this protein helps trigger the release of renin, a hormone that starts a chain reaction known as the renin-angiotensin-aldosterone system, or RAAS. The system is one of the body’s main tools for keeping blood pressure stable and making sure the body has the right balance of salt and water.

Nasal drops fight brain tumors noninvasively

Researchers at WashU Medicine have developed a noninvasive medicine delivered through the nose that successfully eliminated deadly brain tumors in mice. The medicine is based on a spherical nucleic acid, a nanomaterial (labeled red) that travels along a nerve (green) from the nose to the brain, where it triggers an immune response to eliminate the tumor.
Image Credit: Courtesy of Alexander Stegh

Researchers at Washington University School of Medicine in St. Louis, along with collaborators at Northwestern University, have developed a noninvasive approach to treat one of the most aggressive and deadly brain cancers. Their technology uses precisely engineered structures assembled from nano-size materials to deliver potent tumor-fighting medicine to the brain through nasal drops. The novel delivery method is less invasive than similar treatments in development and was shown in mice to effectively treat glioblastoma by boosting the brain’s immune response.

Glioblastoma tumors form from brain cells called astrocytes and are the most common kind of brain cancer, affecting roughly three in 100,000 people in the U.S. Glioblastoma generally progresses very quickly and is almost always fatal. There are no curative treatments for the disease, in part because delivering medicines to the brain remains extremely challenging.

What Is: Mitochondrion

Evolutionary Singularities and the Eukaryotic Dawn

The mitochondrion represents a biological singularity, a discrete evolutionary event that fundamentally partitioned life on Earth into two distinct energetic stratums: the prokaryotic and the eukaryotic. While colloquially reduced to the moniker of "cellular powerhouse," the mitochondrion is, in functional reality, a highly integrated endosymbiont that serves as the master regulator of eukaryotic physiology. It is the nexus of cellular respiration, the arbiter of programmed cell death, a buffer for intracellular calcium, and a hub for biosynthetic pathways ranging from heme synthesis to steroidogenesis. To comprehend the complexity of multicellular life, one must first dissect the intricate molecular sociology of this organelle.   

The origin of the mitochondrion is the subject of intense phylogenomic reconstruction. The prevailing consensus, the endosymbiotic theory, posits that the mitochondrion descends from a free-living bacterial ancestor—specifically a lineage within the Alphaproteobacteria—that entered into a symbiotic relationship with a host archaeal cell approximately 1.5 to 2 billion years ago. This was not a trivial acquisition but a transformative merger. The energetic capacity afforded by the internalization of a bioenergetic specialist allowed the host cell to escape the surface-area-to-volume constraints that limit prokaryotic genome size, facilitating the expansion of the nuclear genome and the development of complex intracellular compartmentalization. 

New type of DNA damage found in our cells’ powerhouses

Linlin Zhao (left) and Yu Hsuan Chen
Photo Credit: Courtesy of University of California, Riverside

A previously unknown type of DNA damage in the mitochondria, the tiny power plants inside our cells, could shed light on how our bodies sense and respond to stress. The findings of the UC Riverside-led study are published in the Proceedings of the National Academy of Sciences and have potential implications for a range of mitochondrial dysfunction-associated diseases, including cancer and diabetes. 

Mitochondria have their own genetic material, known as mitochondrial DNA (mtDNA), which is essential for producing the energy that powers our bodies and sending signals within and outside cells. While it has long been known that mtDNA is prone to damage, scientists didn't fully understand the biological processes. The new research identifies a culprit: glutathionylated DNA (GSH-DNA) adducts.

An adduct is bulky chemical tag formed when a chemical, such as a carcinogen, attaches directly to DNA. If the damage isn’t repaired, it can lead to DNA mutations and increase the risk of disease.

Scientists observe metabolic activity of individual lipid droplets in real time

LipiPB Red shows longer fluorescence lifetimes in stable lipid droplets (red) and shorter lifetimes as they undergo degradation (blue). This probe revealed that lipid droplets sequentially degrade, where lipolysis precedes lipophagy.
Image Credit: Issey Takahashi, Nagoya University

A research team has developed a fluorescent probe that allows scientists to visualize how individual lipid droplets break down inside living cells in real time. The probe changes its fluorescence properties depending on the chemical composition of each droplet, which allows researchers to observe not only their location within cells, but also their metabolic activity during lipid breakdown. The findings, published in the Journal of the American Chemical Society, may contribute to the development of new strategies to treat metabolic diseases such as obesity and diabetes, as well as cancers associated with abnormal lipid metabolism. 

“Lipid droplets are cellular organelles that not only store excess lipids but also play critical roles in lipid metabolism. However, understanding how individual droplets function has been challenging,” Professor Shigehiro Yamaguchi, from the Institute of Transformative Bio-Molecules (ITbM) at Nagoya University, explained. 

A cellular protein, FGD3, boosts breast cancer chemotherapy, immunotherapy

The research team included, front row, from left: graduate student Junyao Zhu, biochemistry professor David Shapiro, and senior researcher Chengiian Mao; back row, from left: graduate students Abigail Spaulding, Xinyi Dai and Qianjin Jiang.
Photo Credit: Fred Zwicky

A naturally occurring protein that tends to be expressed at higher levels in breast cancer cells boosts the effectiveness of some anticancer agents, including doxorubicin, one of the most widely used chemotherapies, and a preclinical drug known as ErSO, researchers report. The protein, FGD3, contributes to the rupture of cancer cells disrupted by these drugs, boosting their effectiveness and enhancing anticancer immunotherapies.

The new findings were the happy result of experiments involving ErSO, an experimental drug that killed 95-100% of estrogen-receptor-positive breast cancer cells in a mouse model of the disease. ErSO upregulates a cellular pathway that normally protects cancer cells from stress, said University of Illinois Urbana-Champaign biochemistry professor David Shapiro, who led the new work with Illinois graduate student Junyao Zhu. But when that protective pathway is ramped up, the system goes awry.

How chromosomes separate accurately

Representation how separase recognizes the cohesin subunit SCC1 before chromosome segregation occurs.
Illustration Credit: © Margot Riggi

Cell division is a process of remarkable precision: during each cycle, the genetic material must be evenly distributed between the two daughter cells. To achieve this, duplicated chromosomes, known as sister chromatids, are temporarily linked by cohesin – a ring-shaped protein complex that holds them together until separation. Researchers at the University of Geneva (UNIGE), in collaboration with the National Cancer Institute (NCI) and the University of California, San Francisco (UCSF), have uncovered the mechanism by which separase – the molecular ‘‘scissors’’ responsible for this cleavage – recognizes and cuts cohesin. Their findings, published in Science Advances, shed new light on chromosome segregation errors that can lead to certain forms of cancer. 

Biochemistry: In-Depth Description

Image Credit: Scientific Frontline

Biochemistry is a branch of biology and chemistry that explores the chemical processes within and relating to living organisms. Its primary goal is to understand the chemical basis of life by studying the structure, function, and interactions of biological macromolecules.

This field seeks to answer fundamental questions about how collections of inanimate molecules interact to constitute, maintain, and perpetuate living organisms.

Nonsurgical treatment shows promise for targeted seizure control

Jerzy Szablowski
Photo Credit: Jeff Fitlow/Rice University

Rice University bioengineers have demonstrated a nonsurgical way to quiet a seizure-relevant brain circuit in an animal model. The team used low-intensity focused ultrasound to briefly open the blood-brain barrier (BBB) in the hippocampus, delivered an engineered gene therapy only to that region and later flipped an on-demand “dimmer switch” with an oral drug. The research shows that a one-time, targeted procedure can modulate a specific brain region without impacting off-target areas of the brain.

“Many neurological diseases are driven by hyperactive cells at a particular location in the brain,” said study lead Jerzy Szablowski, assistant professor of bioengineering and a member of the Rice Neuroengineering Initiative. “Our approach aims the therapy where it is needed and lets you control it when you need it, without surgery and without a permanent implant.”

Scientists Removed Amino Acids From the Diet of Lab Mice — and They Lost Weight

Legumes are a diverse group of plants from the Fabaceae family, including beans, peas, lentils, and peanuts, that grow in pods. They are a highly nutritious food, rich in protein, fiber, vitamins, and minerals, and are often considered a plant-based alternative to animal protein. Legumes also have the unique ability to fix nitrogen from the atmosphere, which benefits soil health.
Photo Credit: Shelley Pauls

It’s not pleasant to shiver from the cold, but for some, it has the appeal of making the body burn more energy as heat than when staying in a warmer environment. According to several studies, exposure to cold is a reliable way to boost energy expenditure in mice and humans. This process of burning energy through heat loss is called thermogenesis.

While scientists and pharmaceutical companies are exploring ways to trick the body into thinking it’s cold—so that it activates thermogenesis and burns energy without the need for ice baths or winter walks in a T-shirt—obesity researchers Philip Ruppert and Jan-Wilhelm Kornfeld from the Department of Biochemistry and Molecular Biology (BMB) set out to investigate another route:

A form of thermogenesis triggered by eating specialized diets rather than temperature.

Researchers decipher mechanism that prevents the loss of brown adipose tissue activity during ageing

From left to right, Tania Quesada-López, Francesc Villarroya, Albert Blasco-Roset, Marta Giralt, Alberto Mestres-Arenas, Joan Villarroya, Aleix Gavaldà-Navarro and Rubén Cereijo.
Photo Credit: Courtesy of University of Barcelona

As the body ages, brown adipose tissue activity decreases, fewer calories are burned, and this can contribute to obesity and certain chronic cardiovascular diseases that worsen with age. A study led by the University of Barcelona has identified a key molecular mechanism in the loss of brown fat activity during ageing. The study opens up new perspectives for designing strategies to boost the activity of this tissue and prevent chronic metabolic and cardiovascular diseases as the population ages.

The paper, published in the journal Science Advances, is led by Professor Joan Villarroya, from the Faculty of Biology and the Institute of Biomedicine of the UB (IBUB) — based at the Barcelona Science Park-UB  — and the CIBER Area for Physiopathology of Obesity and Nutrition  (CIBEROBN). Teams from the Albert Einstein College of Medicine in New York (United States) are also collaborating.

UQ scientists uncover secrets of yellow fever

Dr Summa Bibby
Photo Credit: The University of Queensland

University of Queensland researchers have captured the first high-resolution images of the yellow fever virus (YFV), a potentially deadly viral disease transmitted by mosquitoes that affects the liver.

They’ve revealed structural differences between the vaccine strain (YFV-17D) and the virulent, disease-causing strains of the virus.

Dr Summa Bibby from UQ’s School of Chemistry and Molecular Bioscience said despite decades of research on yellow fever, this was the first time a complete 3D structure of a fully mature yellow fever virus particle had been recorded at near-atomic resolution.

“By utilising the well-established Binjari virus platform developed here at UQ, we combined yellow fever’s structural genes with the backbone of the harmless Binjari virus and produced virus particles that could be safely examined with a cryo-electron microscope,” Dr Bibby said.

Scientists Produce Powerhouse Pigment Behind Octopus Camouflage

An octopus camouflages itself with the seafloor. UC San Diego scientists have discovered a new way to produce large amounts of xanthommatin, a natural pigment used in animal camouflage, in a bacterium for the first time.
Photo Credit: Charlotte Seid

Scientists at UC San Diego have moved one step closer to unlocking a superpower held by some of nature’s greatest “masters of disguise.”

Octopuses, squids, cuttlefish and other animals in the cephalopod family are well known for their ability to camouflage, changing the color of their skin to blend in with the environment. This remarkable display of mimicry is made possible by complex biological processes involving xanthommatin, a natural pigment.

Because of its color-shifting capabilities, xanthommatin has long intrigued scientists and even the military, but has proven difficult to produce and research in the lab — until now.

New switch for programmed cell death identified

During the analysis work: Prof. Franz Hagn (left) and Dr. Umut Günsel
Photo Credit: Astrid Eckert / TUM 

In the fight against disease, programmed cell death – also known as apoptosis – is a key protective function of the body. It breaks down cells that are damaged or have undergone dangerous changes. However, cancer cells often manage to override this mechanism. A research team at the Technical University of Munich (TUM) has now succeeded in identifying a new molecular switch in this process and elucidating how it works.

The activation and deactivation of apoptosis is a promising field of research in basic biomedical research. The team led by Prof. Franz Hagn from the Chair of Structural Membrane Biochemistry at the TUM School of Natural Sciences has now discovered a new switch: "Many research teams worldwide are working on the exciting topic of apoptosis and its targeted control. The big advantage is that we are dealing with a highly efficient, evolutionarily developed regulatory mechanism. So, we don't have to invent something completely new, but can use the appropriate structural methods to learn from nature's optimized processes."