James J. Dowling MD PhD - Professor of Genetics & Neurology
A new study published in Science Translational Medicine highlights findings from researchers at the Perelman School of Medicine at the University of Pennsylvania and collaborating institutions showing that AAV8 gene therapy, when combined with dietary stress, can precipitate cholestatic liver disease in a mouse model of X‑linked myotubular myopathy (XLMTM).This work sheds important light on metabolic factors that influence liver vulnerability during gene therapy and provides new guidance for developing safer therapeutic strategies for rare neuromuscular disorders.The publication led by James J. Dowling and a multi‑institutional team is featured on the cover of the February 18, 2026 issue of Science Translational Medicine.