Split AAV-Mediated Gene Therapy Restores Ureagenesis in a Murine Model of Carbamoyl Phosphate Synthetase 1 Deficiency
Matthew Nitzahn, Gabriella Allegri, Suhail Khoja, Brian Truong, Georgios Makris, Johannes Häberle, Gerald S. Lipshutz
Topics & Concepts
HyperammonemiaUrea cycleLethargyCarbamoyl phosphate synthetaseAtaxiaBiologyPharmacologyInternal medicineMedicineEndocrinologyBiochemistryEnzymeArginineNeuroscienceAmino acidVirus-based gene therapy researchCytomegalovirus and herpesvirus researchRNA Interference and Gene Delivery