Around 10 million people globally live with the life-threatening human T-cell lymphotropic virus type-1 (HTLV-1), yet it remains a poorly understood disease that currently has no preventative treatments and no cure.

The switch will be flicked today to make the world’s largest dementia-related proteomics dataset freely available to researchers, at the same time as members of the consortium which compiled it publish the proteomics signatures of major neurodegenerative diseases that they uncovered in a first trawl of the data.

Lizards, zebrafish, salamanders and tritons can regrow a tail, a fin, or even an entire limb after amputation. Cut a planarian into pieces, and you will end up with a bunch of them. Researchers at the National Institute of Biological Sciences in Beijing have discovered a genetic switch linked to vitamin A. After activating this pathway, they managed to regenerate the ear pinna of a mouse, an animal that previously lacked this ability.

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.

Nuevocor Pte. Ltd. has closed a $45 million series B, enabling it to move lead gene therapy NVC-001 into the clinic in the treatment of an inherited form of cardiomyopathy.

Genes associated with lysosomal dysfunction increase the risk of Parkinson’s disease (PD), according to a study led by scientists at Northwestern University. The discovery also explains why some people who carry a pathogenic variant of the GBA1 gene develop PD or dementia with Lewy bodies (DLB) and others do not. The key lies in the Commander complex, involved in the transport of proteins to this organelle. This discovery raises the need for combinatorial therapies that act on more than one pathway for this type of neurodegenerative disorder.

Researchers have developed a new compound that can prevent long COVID symptoms in mice that could lead to a future drug for the debilitating condition in humans. Developed by researchers at the Walter and Eliza Hall Institute of Medical Research (WEHI) in Melbourne, the world-first study found mice treated with the antiviral compound were protected from long-term brain and lung dysfunction, which are key symptoms of long COVID.

Compared to other forms of prevention, a unique issue for pandemic preparedness is that it is forever unclear what pathogen, exactly, the world needs to be prepared for. There are an estimated 300,000 viruses that infect mammals; add in birds, and the estimate grows to more than half a million. Some of those viruses are much greater threats than others.

Transplanting an animal organ into a human is now a closer reality following the successful xenotransplantation of a genetically modified pig liver into a patient diagnosed with brain death in China. The operation was intended to evaluate organ function over a 10-day period. This is a complex experimental trial that did not involve removing the patient's liver and still requires further study. However, the positive preclinical results suggest this strategy could save the lives of those waiting for a human organ, at least in certain cases.

In 2020, the Conference on Retroviruses and Opportunistic Infections (CROI) was the first scientific conference to move from in-person to virtual due to the COVID-19 pandemic. On the fifth anniversary of the virtual conference, and the pandemic, some of those earliest COVID-19 patients have still not recovered.