Research Highlights
Our research focuses on the development of gene and cell-based therapies for the treatment of metabolic diseases of the liver that commonly result in the development of intellectual disabilities in children. We have four active research directions:
- Development of gene-based (adeno-associated virus, mRNA) therapies to restore nitrogen metabolism, ureagenesis, and creatine biosynthesis.
- Exploring the underlying neurobiology in arginase deficiency and in guanidinoacetate methyltransferase deficiency to gain a greater understanding of the neuropathology that exists in these enzyme deficiencies.
- Developing new therapeutic approaches to treating deficiency of carbamoyl phosphate synthetase 1.
- Understanding the effect of guanidinoacetate on GABA-A receptors in guanidinoacetate methyltransferase deficiency.
Featured Publications
Xu, R., Diep, T., Singh, K., Bakshi, S., Wu, G. F., Lu, M., Tang, G., Schwerk, J., Sun, Q., Häberle, J., Schulze, A., Tkachyova, I., Poms, M., Jacques, B., Rychak, J., Lee, S. J., & Lipshutz, G. S. (2026). A hybrid piggyBac AAV-transposon and LNP-transposase for treating arginase deficiency: A 100-fold dose reduction compared to AAV alone. Mol Ther., S1525-0016(26)00310-2. Advance online publication. https://doi.org/10.1016/j.ymthe.2026.04.045
Diep, T., Zhou, W., Reyes, R.E., Nitzahn, M., Day, I. L., Makris, G., Lueptow, L., Zhuravka, I., Bakshi, S., Gangoiti, J., Padaon, H., Li, Y., Barshop, B., Harberle, J., & Lipshutz, G. S. (2025). Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control. Molecular Therapy – Nucleic Acids, Volume 36, Issue 1. https://doi.org/10.1016/j.omtn.2025.102470
Nitzahn M, Allegri G, Khoja S, Truong B, Makris G, Häberle J, Lipshutz GS. Split AAV-Mediated Gene Therapy Restores Ureagenesis in a Murine Model of Carbamoyl Phosphate Synthetase 1 Deficiency. Mol Ther. 2020 Jul 8;28(7):1717-1730. doi: 10.1016/j.ymthe.2020.04.011.
Recent News
May 18, 2026
The Lipshutz Lab would like to congratulate Rachel Xu, Kiran, and Taryn for their recent publication, “A hybrid piggyBac AAV-transposon and LNP-transposase for treating arginase deficiency: A 100-fold dose reduction... Read More
Sep 11, 2025
Article Link. Congratulations to Dr. Gerry Lipshutz for recently being featured on an article published by UCLA Newsroom titled “A surgeon’s second calling: Gene therapy for the brain’s energy crisis.”... Read More
Oct 31, 2025
The Lipshutz Lab would like to congratulate project scientist Dr. Martin Wallner on his retirement. Thank you for all your contributions to the lab and enjoy retirement life!... Read More
Sep 15, 2025
Congratulations to Taryn for her recent publication with Dr. Gerry Lipshutz titled “Evaluation of SLC6A8 species conservation and the effect of pathogenic variants on creatine transport.” This publication examined evolutionary... Read More