Split AAV-Mediated Gene Therapy Restores Ureagenesis in a Murine Model of Carbamoyl Phosphate Synthetase 1 Deficiency
Nitzahn M, Allegri G, Khoja S, Truong B, Makris G, Häberle J, Lipshutz GS. Split AAV-Mediated Gene Therapy Restores Ureagenesis in a Murine Model of Carbamoyl Phosphate Synthetase 1 Deficiency. Mol Ther. 2020 Jul 8;28(7):1717-1730. doi: 10.1016/j.ymthe.2020.04.011. Epub 2020 Apr 17. PMID: 32359471; PMCID: PMC7335736.