日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Genetic therapy in a mitochondrial disease model suggests a critical role for liver dysfunction in mortality

线粒体疾病模型中的基因治疗表明肝功能障碍在死亡率中起着关键作用

Ankit Sabharwal #, Mark D Wishman #, Roberto Lopez Cervera #, MaKayla R Serres, Jennifer L Anderson, Shannon R Holmberg, Bibekananda Kar, Anthony J Treichel, Noriko Ichino, Weibin Liu, Jingchun Yang, Yonghe Ding, Yun Deng, Jean M Lacey, William J Laxen, Perry R Loken, Devin Oglesbee, Steven A Farber

Characterising a healthy adult with a rare HAO1 knockout to support a therapeutic strategy for primary hyperoxaluria

描述具有罕见 HAO1 基因敲除的健康成年人以支持原发性高草酸尿症的治疗策略

Tracy L McGregor, Karen A Hunt, Elaine Yee, Dan Mason, Paul Nioi, Simina Ticau, Marissa Pelosi, Perry R Loken, Sarah Finer, Deborah A Lawlor, Eric B Fauman, Qin Qin Huang, Christopher J Griffiths, Daniel G MacArthur, Richard C Trembath, Devin Oglesbee, John C Lieske, David V Erbe, John Wright, David