Variant in situ sequencing (VIS-seq) links genetic variants to cell images, revealing how variants affect molecules, subcellular structures, and cells at scale. Applied to thousands of LMNA and PTEN variants, VIS-seq illuminated how variants impacted a multidimensional phenotypic continuum that is not recapitulated by any single functional readout.
A new longevity platform is showing promise across multiple diseases including potential applications in neurologic trauma and coma states. Researchers say the same underlying science could reshape how we think about aging itself.
Heart failure (HF) is a widespread cardiovascular condition that poses significant risks to a wide spectrum of age groups and leads to terminal illness. Although our understanding of the underlying mechanisms of HF has improved, the available treatments still remain inadequate. Recently, long non-coding RNAs (lncRNAs) have emerged as crucial players in cardiac function, showing possibilities as potential targets for HF therapy. These versatile molecules interact with chromatin, proteins, RNA, and DNA, influencing gene regulation. Notable lncRNAs like Fendrr, Trpm3, and Scarb2 have demonstrated therapeutic potential in HF cases.
People living in regions where malaria outbreaks are common experience repeated exposure to the disease, which gradually teaches the body how to fight back. Over time, they develop naturally acquired immunity that helps the body control the density of malaria parasites (Plasmodium falciparum) in the blood and prevent the development of clinical symptoms.
A recent study set out to pinpoint the specific parts of the malaria parasite that the immune system targets to protect the body from disease. The researchers deliberately infected 142 Kenyan adults known to be immune to malaria, then monitored their symptoms and parasite levels. They successfully identified six merozoite antigens —proteins on the surface of the malaria parasite—that were linked to natural immunity against the disease. The findings were published in Nature Communications.
Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer. As published in Science Translational Medicine, the results suggest that a one-time genetic correction could someday treat the root cause of the disease rather than just managing its symptoms. The work represents a major step for genetic medicine, as restoring disease-relevant brain function with gene editing tools remains a major challenge.
The study also reflects growing momentum behind gene editing as a therapeutic platform for rare diseases. In February 2026, the Food and Drug Administration issued its Plausible Mechanism Framework guidance, outlining a regulatory pathway for individualized therapies targeting specific genetic conditions. It recognizes that for rare genetic diseases, a well-characterized biological mechanism can serve as the foundation for approval where large clinical trials are not feasible.
“For families affected by Dravet syndrome, our study provides proof of concept that a genetic correction approach could have real impact, a future with treatments that don’t just manage the disease but actually address its cause,” said Matthew Simon, a senior study director at The Jackson Laboratory (JAX) Rare Disease Translational Center (RDTC) who co-led the study. “We’re at an inflection point in genetic medicine, where we can now actually repair the DNA itself.”
Oxygen is a cornerstone of chemistry, largely because it is so good at building the organic molecules that make up our world. Some oxygen-based compounds called peroxides are famous for being highly reactive—they act like oxygen delivery trucks, transferring atoms to other molecules. This process is essential for everything from creating new medicines to industrial manufacturing.
In a study published in Nature Chemistry, researchers from the labs of MIT professors Christopher C. Cummins and Robert J. Gilliard, Jr. have revealed a brand-new type of peroxide containing boron. This molecule, called a dioxaborirane, represents a major advance in a field where such structures were long-proposed, but considered too unstable to actually isolate.
Immunotherapy targeting programmed cell death protein 1 (PD-1) and programmed death ligand 1 (PD-L1) has transformed the management of several types of cancers, including non-oncogene-addicted non-small cell lung cancer (NSCLC) [1], although its efficacy remains limited by resistance mechanisms and constraints inherent to monoclonal antibodies [1]. To overcome these drawbacks, small-molecule PD-L1 inhibitors have been developed, and we previously contributed by identifying the nanomolar triazine-based ligand Tr-10 [2]. In parallel, combinatorial strategies aimed at improving the efficacy of anti-PD-1/PD-L1 immunotherapy have gained increasing attention. Notably, platinum-based chemotherapy combined with immune checkpoint inhibitors is recommended as a first-line treatment for advanced NSCLC with PD-L1 expression <50% [3]. Here, we investigated a novel combination involving our anti-PD-L1, Tr-10 [2], and a bis(phenyl-pyridine)iridium(III) complex, Ir-2 (Fig. 1A) [4]. Iridium (Ir) complexes, unlike platinum drugs, are chemically inert and induce endoplasmic reticulum (ER) stress and overproduction of reactive oxygen species (ROS) [5,6], both culminating in damage-associated molecular pattern (DAMP) release and immunogenic cell death (ICD). Moreover, their photophysical properties enable PD-L1-targeted bioimaging when coupled with PD-L1 ligands (Fig. S1) [7].
Aging is accompanied by profound alterations in immune function, termed immunosenescence, and by a chronic, low-grade inflammatory state known as inflammaging. These processes are increasingly recognized as central drivers of age-related neurodegenerative diseases, including Alzheimer’s Disease, Parkinson’s Disease, Amyotrophic Lateral Sclerosis and Multiple Sclerosis. In the central nervous system, senescent microglia and astrocytes lose their homeostatic and neuroprotective functions, while systemic immune aging and blood–brain barrier dysfunction further amplify neuroinflammation and impair protein aggregate clearance. This sustained pro-inflammatory environment promotes synaptic dysfunction, neuronal loss and cognitive decline.