Researchers have identified KpsM as a virulence factor in Escherichia coli that was responsible for liver damage in alcohol-associated hepatitis (AH). A small-molecule inhibitor of KpsM reduced liver damage in animal models of AH.

Individuals with both sickle cell disease (SCD) and sickle cell trait are at higher risk than others of developing renal medullary cancer (RMC), the rarest and deadliest subtype of kidney cancer. Researchers at MD Anderson Cancer Center have identified the molecular mechanisms behind the increased risk, gaining new insights into antitumor immunity more generally and, potentially, new ways to treat RMC, and possibly other tumors as well.

SCD “has been studied for 30 years, but 95% of the effort [has been] working on the red blood cells … how red blood cells contribute to hypoxia and then reduce oxygen supply,” Chunru Lin told BioWorld.

Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.

Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.

Immunity is not a function most people particularly associate with the liver. But because of its connection to the gut, the liver is exposed to bacterial metabolites as few other organs are. And when either the liver or the gut is not functioning well, it can adversely affect immunity as well.

Immunity is not a function most people particularly associate with the liver. But because of its connection to the gut, the liver is exposed to bacterial metabolites as few other organs are. And when either the liver or the gut is not functioning well, it can adversely affect immunity as well. The liver is connected to the gut via both the biliary system and the portal vein. Those two conduits allow metabolites from the gut microbiome to influence what’s going on in the liver. Both liver and gut damage can affect this communication for the worse. And surprisingly, one of the consequences is immune dysfunction.

“I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”

Targeted protein degradation has yet to notch its first approval. But with more than two dozen agents now in clinical trials, the strategy’s ultimate clinical validation appears to be a matter of time.

“I think we’ve come a long way in understanding the importance of this biology. We know it affects men and women, children and adults,” Paul Mischel told the audience during his plenary talk at the 2025 Annual Meeting of the American Association for Cancer Research (AACR 2025). “It’s very prevalent, it’s very devastating. It creates resistance. And we’ve learned some very fundamental rules about this biology that are driving it.”

“Just simply getting old, from age 50 to 75, increased risk for Alzheimer’s disease by 100-fold, which really dwarfed 10-fold increase in risk, conferred by all known risk factors combined, including APOE genotype, being a female, hypertension, smoking, physical inactivity and diabetes.