Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control.
使用超大的 AAV8 载体治疗 CPS1 缺陷可实现长期存活和氨控制
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102470
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
