A new gene therapy has been used to successfully treat a deadly childhood liver disease in mice that model the disease, according to researchers at UCL and Great Ormond Street Hospital. Arthrogryposis, renal dysfunction and cholestasis (ARC) syndrome is a lethal genetic disorder usually caused by a lack of the VPS33B protein, with children diagnosed with the condition rarely living beyond their first year of life.

Now, in a study published in Nature Communications, the UCL-GOSH team found that by injecting a healthy version of the gene into the body, they can treat the condition in mice lacking VPS33B. Crucially, the final version of the treatment, which specifically targeted the liver cells, caused no harm. In the earlier versions, the genes became abnormally activated and caused cancerous cells to grow and expand in some cases.

While more tests must be done before the treatment can be tested in humans, the researchers’ breakthrough offers hope to babies with this devastating disorder and their families. In the UK, as many as six pregnancies per year might be affected by ARC syndrome. Furthermore, the findings may promote improved understanding of why some treatments may cause cancer.

What if a single sentence could carry two completely different meanings, one when read forward and another when read backward? In a new study, researchers at Arizona State University have discovered a biological version of this idea. Working with the mitochondria of a tiny insect called the citrus mealybug, the team found that the same stretch of DNA can carry two different genes—sets of genetic instructions used by the cell—with one encoded on each strand of the DNA’s ladder-like structure.

The finding expands scientists’ understanding of how DNA can store genetic information and helps solve a mystery that has puzzled researchers for years. The findings are published in the journal Proceedings of the National Academy of Sciences.

“This kind of paper is what makes running a lab so fun. Born from a spark of individual brilliance—not mine—but accomplished as a collective effort,” says John McCutcheon. “The idea that these two critically important genes could be mirrored on the same piece of DNA has been around a long time, and so it’s a thrill to be part of the team that proved this speculative idea was, in fact, reality.”

Red blood cell (RBC) production, or erythropoiesis, serves as a paradigm for studying cellular differentiation in both physiological and pathological contexts. While the transcriptional and epigenetic programs controlling erythropoiesis are well characterized, the metabolic regulation of this complex process remains underexplored. Recent discoveries that pyruvate kinase activators improve outcomes in sickle cell disease and thalassemia underscore the therapeutic potential of targeting metabolism in RBC disorders. However, further progress is limited by an incomplete understanding of the metabolic networks supporting erythropoiesis and RBC function.

A comprehensive multi-cancer study by researchers at The University of Texas MD Anderson Cancer Center has revealed that cancer cells within tumors are genetically diverse, yet all carry the same core genetic changes that can be traced back to a common ancestral cell, providing a single-cell view of how tumors adapt, survive and diversify. Understanding this helps explain why some cancer cells manage to survive treatment, paving the way for more tailored diagnostic and therapeutic strategies.

The study, published in Cancer Discovery, was led by Nicholas Navin, Ph.D., chair of Systems Biology. The research shows that cancer cells do not evolve slowly over time, but rather grow through sudden bursts of rapid genetic changes that include copy number alterations (CNAs)—gains or losses of entire sections of DNA. This creates a family tree of distinct new subpopulations that can influence tumor aggressiveness, metastasis and treatment response.

“Our findings provide the clearest views to date of how cancers originate and evolve at the single-cell level,” Navin said. “By revealing both the shared early genetic events and the bursts that drive ongoing diversity, we now have a roadmap for developing smarter clinical diagnostic and treatment strategies to improve patient outcomes.”