A hybrid piggyBac AAV-transposon and LNP-transposase for treating arginase deficiency: A 100-fold dose reduction compared to AAV alone
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. Molecular therapy : the journal of the American Society of Gene Therapy, S1525-0016(26)00310-2. Advance online publication. https://doi.org/10.1016/j.ymthe.2026.04.045
