From small ions to large molecules, cellular gates control what can pass in and out of cells. But how one such gate, called pannexin-1 (PANX1), can handle vastly different cargo sizes has remained a long-standing mystery.

In a new study, Northwestern University scientists uncovered the molecular trick behind PANX1’s versatility.

A new multi-site study led by researchers at CU Anschutz shows that people with cystic fibrosis (CF) who start the triple-drug therapy elexacaftor/tezacaftor/ivacaftor (ETI) can safely reduce many of their daily lung treatments while maintaining good health for years.

The study was published in the Journal of Cystic Fibrosis.

For the first time, MIT chemists have synthesized a fungal compound known as verticillin A, which was discovered more than 50 years ago and has shown potential as an anticancer agent.

The compound has a complex structure that made it more difficult to synthesize than related compounds, even though it differed by only a couple of atoms.

MA-5 is a therapeutic intervention for mitochondrial disease that has been demonstrated to ameliorate severe symptoms such as neurological disorders, renal dysfunction, hepatic dysfunction and motor disorders in animal disease models, which are induced by mitochondrial dysfunction. Crucially, it also improved survival rates.

Researchers at The University of Texas MD Anderson Cancer Center have identified a specific protein, RASH3D19, that is responsible for activation of RAS signaling pathways involved in aggressive tumor growth and resistance to KRAS inhibitors in patients with KRAS-mutant cancers. Blocking RASH3D19 in combination with KRAS inhibitors improved outcomes in preclinical models, suggesting this combination as a potential therapeutic strategy for patients with KRAS-mutant cancers.

Cedars-Sinai scientists have developed an experimental drug that repairs DNA and serves as a prototype for a new class of medications that fix tissue damage caused by heart attack, inflammatory disease or other conditions.

Investigators describe the workings of the drug, called TY1, in a paper published in Science Translational Medicine.

Finland was the first country to offer the zoonotic avian influenza A(H5N8) vaccine manufactured by Seqirus to at-risk occupational groups following the extensive clade 2.3.4.4b A(H5N1) outbreak affecting wild birds and fur farms in Finland in 2023.

A new study published in Nature Microbiology shows that the MF59-adjuvanted A(H5N8) vaccine induced strong immune responses, including both functional antibodies and memory T-cell responses,

The human P2X4 receptor plays an important role in chronic pain, inflammation and some types of cancer. Researchers at the University of Bonn and the University Hospital Bonn (UKB) have now discovered a mechanism that can inhibit this receptor. The results were recently published in the scientific journal Nature Communications and open up a pathway for the development of new drugs.

After a spinal cord injury, cells in the brain and spinal cord change to cope with stress and repair tissue. A new study from Karolinska Institutet, published in Nature Neuroscience, shows that this response is controlled by specific DNA sequences. This knowledge could help develop more targeted treatments.

When the central nervous system is damaged - for example, in a spinal cord injury - many cells become reactive.

Protein kinases are the molecular switches of the cell. They control growth, division, communication, and survival by attaching phosphate groups to other proteins. When these switches are stuck in the "on" position, they can drive cancer and other diseases. Not surprisingly, kinases have become one of the most important drug target families in modern medicine: today, more than 80 kinase inhibitors are FDA-approved, and nearly twice as many are in clinical development.

The U.S. Food and Drug Administration today announced the deployment of agentic AI capabilities for all agency employees. Agentic AI capabilities will enable the creation of more complex AI workflows - harnessing various AI models - to assist with multi-step tasks.

Agentic AI refers to advanced artificial intelligence systems designed to achieve specific goals by planning, reasoning, and executing multi-step actions.

Researchers at the Icahn School of Medicine at Mount Sinai and collaborators have reported early success with a novel mRNA-based therapy designed to combat antibiotic-resistant bacteria.

The findings, published in the November 26 online issue of Nature Biotechnology, show that in preclinical studies in mice and human lung tissue in the lab, the therapy slowed bacterial growth, strengthened immune cell activity, and reduced lung tissue damage in models of multidrug-resistant pneumonia.

A Garvan-led clinical trial has found that using a common and inexpensive type 2 diabetes drug reduces insulin needs in type 1 diabetes, opening doors for improved management of the condition.

For years doctors have prescribed metformin, an old but common type 2 diabetes medication, to treat insulin resistance in type 1 diabetes. This has been largely based on anecdotal evidence.

Ageing is defined as the deterioration of function overtime, and it is one of the main risk factors for numerous chronic diseases. Although ageing is a complex phenomenon affecting the whole organism, it is proved that the solely manifestation of ageing in the haematopoietic system affects the whole organism. Last September, Dr. M. Carolina Florian and her team revealed the significancy of using blood stem cells to pharmacologically target ageing of the whole body, thereby suggesting rejuvenating strategies that could extend healthspan and lifespan.

Rutgers neuroscientist Peng Jiang was visiting his hometown of Qianshan, a city in China’s Anhui province, when a neighbor came to his parents’ house with a story that would stay with him.

The man’s mother had been diagnosed with Alzheimer’s disease in her early 60s. After nearly a decade of decline, she no longer recognized her own son. One morning, she looked at him and asked gently, "How is your mother doing? Is she well?"

Researchers at the Paul Scherrer Institute PSI have clarified how spermine - a small molecule that regulates many processes in the body's cells - can guard against diseases such as Alzheimer's and Parkinson's: it renders certain proteins harmless by acting a bit like cheese on noodles, making them clump together. This discovery could help combat such diseases.

Cancer research, drug safety testing and ageing biology may all gain a major boost from a new fluorescent sensor developed at Utrecht University. This new tool allows scientists to watch DNA damage and repair unfold in real time inside living cells. The development, which opens the door to experiments that weren't feasible before, is published in the journal Nature Communications.

A new study from the Systems Immunology Research Group at the Institute of Biochemistry, HUN-REN Biological Research Centre, Szeged, reveals that our immune system does more than defend against viruses. In certain cases, it induces mutations within viruses that make them easier to recognize later on. The findings have been published in Nature Communications.

A collaborative research team, led by scientists at VCU Massey Comprehensive Cancer Center and Huntsman Cancer Institute at the University of Utah, has identified a targeted drug that could effectively treat an aggressive soft tissue cancer that occurs most often in pediatric and young adult patients. New findings - published in Nature Communications - suggest that blocking a specific genetic pathway could reverse a series of tumor-driving cellular interactions

The age-old advice to “trust your gut” could soon take on new meaning for people diagnosed with Parkinson’s Disease, thanks to a creative feat of bioengineering by researchers in the University of Georgia’s College of Veterinary Medicine.

Anumantha Kanthasamy, professor and director of the Isakson Center for Neurological Disease Research (ICNDR) leads a multidisciplinary research team including Gregory Phillips, Piyush Padhi, and other scientists that has engineered a groundbreaking living medicine, a beneficial probiotic designed to deliver Levodopa steadily from the gut to the brain of Parkinson's patients.