Matt is using iPSCs and viruses to investigate gene therapy strategies for congenital liver metabolism disorders.
Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice – Stephanie A.K. Angarita, Brian Truong, Suhail Khoja, Matthew Nitzahn, Abha K. Rajbhandari, Irina Zhuravka, Sergio Duarte, Michael G. Lin, Alex K. Lam, Stephen D. Cederbaum, Gerald S Lipshutz. Molecular Genetics and Metabolism 2018 https://doi.org/10.1016/j.ymgme.2018.04.005
Conditional Disruption of Hepatic Carbamyl Phosphate Synthetase 1 in Mice Results in Hyperammonemia without Orotic Aciduria – Suhail Khoja, Matthew Nitzahn, Kip Hermann, Brian Truong, Roberta Borzone, Brandon Willis, Mitchell Rudd, Donna J. Palmer, Philip Ng, Nicola Brunetti-Pierri, Gerald S. Lipshutz. Molecular Genetics and Metabolism 2018 https://doi.org/10.1016/j.ymgme.2018.04.005
Poster presenter at the 21st annual conference for the American Society of Gene and Cell Therapy. “Split AAV-Mediated Gene Addition Therapy for Carbamoyl Phosphate Synthetase 1 Deficiency.”