Overview

• Penn Vet and CHOP researchers report that mitochondrial deficits in blood–brain barrier endothelial cells contribute to barrier leakage in 22q11.2 deletion syndrome.
• Using patient-derived iPSC brain endothelial cells and an animal model, the team found a compromised barrier consistent with a “leaky” BBB.
• Treatment with bezafibrate improved mitochondrial measures and restored barrier integrity in both cellular and preclinical systems.
• In the animal model, bezafibrate also corrected a social memory deficit linked to BBB dysfunction and relevant to schizophrenia.
• Published in Science Translational Medicine, the work suggests potential drug repurposing for 22q11.2 deletion syndrome and possible relevance to psychosis beyond this disorder, pending clinical trials.