Gene editing can repair mutations that prematurely halt protein synthesis, resulting in incomplete peptides that cause various diseases. However, other approaches achieve the same effect without altering the genome. Startup Alltrna Inc. has developed a strategy based on transfer RNA to bypass the premature stop codons that end early protein translation. The company already has a first clinical candidate that could treat metabolic diseases such as methylmalonemia or phenylketonuria.

Advances in antiretroviral therapy (ART) now allow people living with HIV to lead normal lives with undetectable and nontransmissible levels of the virus in their blood. Yet that reality is limited to those with access to treatment. More than 40 million people worldwide live with HIV, with over a million new infections and hundreds of thousands of deaths each year, underscoring that major challenges remain.

The cardiomyositis that is a rare adverse effect of mRNA-based COVID vaccines is due to immune cell activity as a result of increased levels of the chemokines CXCL10 and interferon-γ (IFN-γ). Blocking CXCL10 and IFN-γ could prevent muscle cell damage in cell culture, and cardiomyositis in animal models. The findings, reported in the Dec. 10, 2025, issue of Science Translational Medicine, suggest a way of mitigating the risk of cardiomyositis.