Rescue of multiple class II CFTR mutations by elexacaftor+tezacaftor+ivacaftor mediated in part by the dual activities of elexacaftor as both corrector and potentiator

por Laselva, Onofrio, Bartlett, Claire, Gunawardena, Tarini N.A., Ouyang, Hong, Eckford, Paul D.W., Moraes, Theo J., Bear, Christine E., Gonska, Tanja
Publicado 2021

A new platform for high-throughput therapy testing on iPSC-derived lung progenitor cells from cystic fibrosis patients

por Jiang, Jia Xin, Wellhauser, Leigh, Laselva, Onofrio, Utkina, Irina, Bozoky, Zoltan, Gunawardena, Tarini, Ngan, Zoe, Xia, Sunny, Di Paola, Michelle, Eckford, Paul D.W., Ratjen, Felix, Moraes, Theo J., Parkinson, John, Wong, Amy P., Bear, Christine E.
Publicado 2021

Orkambi® and amplifier co‐therapy improves function from a rare CFTR mutation in gene‐edited cells and patient tissue

por Molinski, Steven V, Ahmadi, Saumel, Ip, Wan, Ouyang, Hong, Villella, Adriana, Miller, John P, Lee, Po‐Shun, Kulleperuma, Kethika, Du, Kai, Di Paola, Michelle, Eckford, Paul DW, Laselva, Onofrio, Huan, Ling Jun, Wellhauser, Leigh, Li, Ellen, Ray, Peter N, Pomès, Régis, Moraes, Theo J, Gonska, Tanja, Ratjen, Felix, Bear, Christine E
Publicado 2017