Matthew Nitzahn


Research project:

Matt is using iPSCs and viruses to investigate gene therapy strategies for congenital liver metabolism disorders.


Split AAV-Mediated Gene Therapy Restores Ureagenesis in a Murine Model of Carbamoyl Phosphate Synthetase 1 DeficiencyNitzahn M, Allegri G, Khoja S, Truong B, Makris G, Häberle J, Lipshutz GS. Molecular Therapy. 2020 Jul 8;28(7):1717–1730.

A constitutive knockout of murine carbamoyl phosphate synthetase 1 results in death with marked hyperglutaminemia and hyperammonemia – Khoja S, Nitzahn M, Truong B, Lambert J, Willis B, Allegri G, Rufenacht V, Haberle J, Lipshutz G. Journal of Inherited Metabolic Diseases.

Hepatic glutamine synthetase augmentation enhances ammonia detoxification – Soria LR, Nitzahn M, De Angelis A, Khoja S, Attanasio S, Annunziata P, Palmer DJ, Ng P, Lipshutz GS, Brunetti-Pierri N. Journal of Inherited Metabolic Disease.

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.

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.


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.”