➀ Researchers at the University of Pennsylvania and Moderna have developed a lipid nanoparticle-encapsulated mRNA therapy that significantly improves survival and reduces leucine levels in a mouse model of maple syrup urine disease (MSUD). ➁ The treatment strategy considers all genetic mutations causing MSUD and shows potential as a long-term universal treatment approach. ➂ Additionally, the Wilson lab has discovered novel AAV variants with improved biodistribution properties, potentially enhancing gene therapy targeting tissues other than the liver.

1. Researchers at Vanderbilt University, led by John T. Wilson, have developed lipid nanoparticles that stimulate the immune system to fight cancer in mice. 2. These nanoparticles transport a nucleic acid molecule that activates the RIG-I pathway, commonly used to detect foreign viruses and aid immune response. 3. The technology shows promise in mouse models of breast cancer and melanoma, with potential for future clinical testing in humans.