JAX research roundup: virtual hearts, flu therapy, and more

Beating heart toxicity at its core: AI-powered “virtual hearts” offer a safer path for new therapies

Developing new drugs is one of the riskiest, slowest and most expensive challenges in medicine. Despite passing early safety standards, more than 90% of drug candidates fail—often due to dangerous side effects that don’t appear until late in clinical trials. To meet this challenge, JAX has launched a new project called CARDIOVERSE, which aims to revolutionize drug safety testing with AI-powered “virtual hearts.” CARDIOVERSE will leverage mouse models, human stem cells, and advanced AI to create computational “digital twins” of the human heart. By building these virtual, beating hearts, scientists aim to dramatically reduce the need for large-animal studies, streamline FDA approval, and ensure safer, faster delivery of new therapies to patients. This exciting new project is powered by an up to $30 million award from the Advanced Research Projects Agency for Health (ARPA-H).

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JAX plays key role in national effort to decode the function of every human gene

More than two decades after the human genome was first sequenced, scientists still don't know what many of its approximately 20,000 protein-coding genes actually do. While a handful of genes have been intensely studied, most remain biological mysteries, leaving major gaps in the understanding of how human biology works and how it goes wrong in disease. By systematically disrupting genes in human cells, JAX researchers are helping to build the first comprehensive atlas of gene function—revealing insights into development, disease, and the future of precision medicine. Backed by the National Human Genome Research Institute, MorPhiC (short for Molecular Phenotypes of Null Alleles in Cells) is a nationwide initiative to determine the function of every human gene by “turning off” each one and seeing how living cells respond.

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Could routine eye exams reveal early signs of Alzheimer’s?

Research by JAX scientists has strengthened the link between retinal health and early dementia risk. The findings, which built on previous work from the same group in the Howell Lab, linked abnormal changes in the tiny blood vessels of the retinas of mice with a common genetic mutation known to increase Alzheimer’s disease risk. Because the retina is part of the central nervous system, scientists often see it as an extension of the brain that shares essentially the same tissue. Changes in retinal blood vessels could offer early clues about brain health. Within the next few years, doctors may be able to spot signs of Alzheimer’s disease and other dementias using routine eye exams well before symptoms appear.

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A ‘universal’ therapy against the seasonal flu? Antibody cocktail targets virus weak spot

An unusual therapy developed at JAX could change the way the world fights influenza, one of the deadliest infectious diseases. Researchers in the Paust Lab reported that a cocktail of antibodies protected mice—including those with weakened immune systems—from nearly every strain of influenza tested, including avian and swine variants that pose pandemic threats. Unlike current FDA-approved flu treatments, which target viral enzymes and can quickly become useless as the virus mutates, this therapy did not allow viral escape, even after a month of repeated exposure in animals. That difference could prove crucial in future outbreaks, when survival often depends on how quickly and effectively doctors can deploy treatments and vaccine development takes months.

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Study uncovers molecular “switch” behind chemoresistance in blood cancer to chemotherapy in acute myeloid leukemia.

One of the biggest challenges in cancer treatment is that certain cancers reappear after chemotherapy. Acute myeloid leukemia (AML), an aggressive type of blood cancer, is notorious for this. Research from the Wang Lab, points to a previously unknown molecular mechanism behind that chemoresistance, and a way to potentially disarm it. By studying data from AML patients from before they received chemotherapy and again after their cancer returned, the team found that in many cases a chemical tag known as DNA methylation had appeared in a section of the genome that normally controls the RUNX1 gene. That small change flipped a genetic switch, forcing cancer cells to make more of the RUNX1C variant of the gene, activating a mechanism that made them far better at withstanding chemotherapy.

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