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

por McGregor, Tracy L, Hunt, Karen A, Yee, Elaine, Mason, Dan, Nioi, Paul, Ticau, Simina, Pelosi, Marissa, Loken, Perry R, Finer, Sarah, Lawlor, Deborah A, Fauman, Eric B, Huang, Qin Qin, Griffiths, Christopher J, MacArthur, Daniel G, Trembath, Richard C, Oglesbee, Devin, Lieske, John C, Erbe, David V, Wright, John, van Heel, David A
Publicado 2020

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

por Sabharwal, Ankit, Wishman, Mark D, Cervera, Roberto Lopez, Serres, MaKayla R, Anderson, Jennifer L, Holmberg, Shannon R, Kar, Bibekananda, Treichel, Anthony J, Ichino, Noriko, Liu, Weibin, Yang, Jingchun, Ding, Yonghe, Deng, Yun, Lacey, Jean M, Laxen, William J, Loken, Perry R, Oglesbee, Devin, Farber, Steven A, Clark, Karl J, Xu, Xiaolei, Ekker, Stephen C
Publicado 2022