Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control
Titel:
Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control
Auteur:
Diep, Taryn Zhou, Wesley Reyes, Rachel E. Nitzahn, Matthew Day, Isabel L. Makris, Georgios Lueptow, Lindsay Zhuravka, Irina Bakshi, Stuti Gangoiti, Jon Padaon, Hyacinth Li, Yunfeng Barshop, Bruce A. Haberle, Johannes Lipshutz, Gerald S.