Use of an Oversized AAV8 Vector for CPS1 Deficiency Results in Long-Term Survival and Ammonia Control

Wesley Zhou, T. Diep, M. Nitzahn, I. Day, P. Patel, G. Makris, L. Lueptow, I. Zhuravka, J. Gangoiti, H. Padaon,B. Barshop,J. Haberle,Gerald S. Lipshutz

MOLECULAR THERAPY（2024）

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MT: Delivery Strategies,gene therapy,carbamoyl phosphate synthetase deficiency,oversized AAV,ureagenesis,hyperammonemia

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