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Split AAV-Mediated Gene Therapy Restores Ureagenesis in a Murine Model of Carbamoyl Phosphate Synthetase 1 Deficiency
Affiliation:1. Molecular Biology Institute, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA;2. Department of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA;3. Division of Metabolism and Children’s Research Center, University Children’s Hospital, Zurich, Switzerland;4. Molecular and Medical Pharmacology, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA;5. Department of Psychiatry, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA;6. Intellectual and Developmental Disabilities Research Center, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA;7. Semel Institute for Neuroscience, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, USA
Abstract:
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  • Keywords:carbamoyl phosphate synthetase deficiency  ureagenesis  hyperammonemia  gene therapy  adeno-associated virus  urea cycle disorder  split AAV
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