Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control
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