ORKAMBI/amplifier co-therapy rescues a rare CFTR mutation in gene-edited cells and patient tissue

MOLINSKI S.;AHMADI S.;KULLEPERUMA K.;IP W.;OUYANG H.;VILLELLA A.;MILLER J. P.;LEE P. S;KULLEPERUMA K.;DU K;DI PAOLA M.;ECKFORD P.;LASELVA O.;HUAN L. J.;WELLHAUSER L;LI E.;RAY P. N;MORAES T. J. GONSKA T.;RATJEN F;BEAR C. E

2017-01-01