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

Suhail Khoja(University of California, Los Angeles), Matthew Nitzahn(University of California, Los Angeles), Kip Hermann(University of California, Los Angeles), Brian Truong(University of California, Los Angeles), Roberta Borzone(Telethon Institute Of Genetics And Medicine), Brandon Willis(University of California, Davis), Mitchell Rudd(University of California, Los Angeles), Donna Palmer(Baylor College of Medicine), Philip Ng(Baylor College of Medicine), Nicola Brunetti‐Pierri(Telethon Institute Of Genetics And Medicine), Gerald S. Lipshutz(University of California, Los Angeles)
Molecular Genetics and Metabolism
April 11, 2018
10.1016/j.ymgme.2018.04.001
Cited by 24Open Access
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