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Molecular Genetics and Metabolism|May 27, 2018
Conditional disruption of hepatic carbamoyl phosphate synthetase 1 in mice results in hyperammonemia without orotic aciduria and can be corrected by liver-directed gene therapySuhail Khoja, Matt Nitzahn, Kip Hermann, et al.
Kidney International|May 30, 2013
Kidney paired donation in the presence of donor-specific antibodiesJeremy M Blumberg, Hans A Gritsch, Elaine F Reed, et al.
JCI Insight|September 6, 2019
Hepatic arginase deficiency fosters dysmyelination during postnatal CNS developmentXiao-Bo Liu, Jillian R Haney, Gloria Cantero, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 4, 2014
Myocyte-mediated arginase expression controls hyperargininemia but not hyperammonemia in arginase-deficient miceChuhong Hu, Jennifer Kasten, Hana Park, et al.
Stem Cells Translational Medicine|February 8, 2017
Functional Human and Murine Tissue-Engineered Liver Is Generated from Adult Stem/Progenitor CellsNirmala Mavila, Andrew Trecartin, Ryan Spurrier, et al.
Nature|April 3, 2024
Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemiaDwight Koeberl, Andreas Schulze, Neal Sondheimer, et al.
Molecular Therapy. Methods & Clinical Development|August 9, 2016
Investigating the functionality of an OCT4-short response element in human induced pluripotent stem cellsAgustin Vega-Crespo, Brian Truong, Kip J Hermann, et al.
Molecular Genetics and Metabolism|May 5, 2018
Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in miceStephanie A K Angarita, Brian Truong, Suhail Khoja, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 16, 2009
Short-term correction of arginase deficiency in a neonatal murine model with a helper-dependent adenoviral vectorChia-Ling Gau, Robin A Rosenblatt, Vincenzo Cerullo, et al.
Molecular Genetics and Metabolism|August 8, 2013
Lethal phenotype in conditional late-onset arginase 1 deficiency in the mouseJennifer Kasten, Chuhong Hu, Ragini Bhargava, et al.
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