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Conditional disruption of hepatic carbamoyl phosphate synthetase 1 in mice results in hyperammonemia without orotic aciduria and can be corrected by liver-directed gene therapy.
Khoja, Suhail; Nitzahn, Matt; Hermann, Kip; Truong, Brian; Borzone, Roberta; Willis, Brandon; Rudd, Mitchell; Palmer, Donna J; Ng, Philip; Brunetti-Pierri, Nicola; Lipshutz, Gerald S.
Affiliation
  • Khoja S; Departments of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, United States.
  • Nitzahn M; Departments of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, United States; Molecular Biology Institute, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, United States.
  • Hermann K; Departments of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, United States; Molecular and Medical Pharmacology, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, United States.
  • Truong B; Departments of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, United States; Molecular and Medical Pharmacology, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, United States.
  • Borzone R; Telethon Institute of Genetics and Medicine, Naples, Italy.
  • Willis B; Mouse Biology Program (MBP), University of California, Davis, United States.
  • Rudd M; Departments of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, United States.
  • Palmer DJ; Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, United States.
  • Ng P; Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, United States.
  • Brunetti-Pierri N; Telethon Institute of Genetics and Medicine, Naples, Italy; Department of Translational Medicine, Federico II University of Naples, Naples, Italy.
  • Lipshutz GS; Departments of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, United States; Molecular and Medical Pharmacology, David Geffen School of Medicine at UCLA, Los Angeles, CA 90095, United States; Molecular Biology Institute, David Geffen School of Medicine at UCLA, Los Angeles,
Mol Genet Metab ; 124(4): 243-253, 2018 08.
Article in En | MEDLINE | ID: mdl-29801986
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Carbamoyl-Phosphate Synthase (Ammonia) / Genetic Therapy / Carbamoyl-Phosphate Synthase I Deficiency Disease / Hyperammonemia Limits: Animals / Female / Humans / Male Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2018 Document type: Article Affiliation country: Estados Unidos Country of publication: Estados Unidos
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Carbamoyl-Phosphate Synthase (Ammonia) / Genetic Therapy / Carbamoyl-Phosphate Synthase I Deficiency Disease / Hyperammonemia Limits: Animals / Female / Humans / Male Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2018 Document type: Article Affiliation country: Estados Unidos Country of publication: Estados Unidos