AAV-mediated gene therapy in a model of SLC13A5 citrate transporter disorder rescues epileptic and metabolic phenotypes.
在 SLC13A5 柠檬酸转运蛋白疾病模型中,AAV 介导的基因治疗可挽救癫痫和代谢表型。
期刊:Journal of Clinical Investigation
影响因子:13.6
doi:10.1172/JCI197503
Bailey Lauren E, Adams Raegan M, Schackmuth Morgan K, Garza Irvin T, Knight Krishanna, Holmes Sydni K, Eller Meghan M, Lee MinJae, Bailey Rachel M