The Odd Couple: Replacement Therapy for Citrate Transporter Deficiency in the Brain and the Liver
AAV-mediated gene therapy in a model of SLC13A5 citrate transporter disorder rescues epileptic and metabolic phenotypes Bailey LE, Adams RM, Schackmuth MK, Garza IT, Knight K, Holmes SK, Eller MM, Lee M, and Bailey RM. J Clin Invest. 2026 Feb 19;136(8):e197503. doi: 10.1172/JCI197503. PMID: 41712282; PMCID: PMC13078891...