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.