Experimental gene therapy targets rare metabolic disease in toddlers

This early-stage trial tests a gene therapy for propionic acidemia, a rare genetic disorder that causes dangerous metabolic crises and organ damage. The study will enroll 9 children aged 6 months to 2 years with confirmed PCCA gene mutations. The therapy uses a harmless virus to deliver a working copy of the gene, aiming to reduce toxic buildup and improve health.