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

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

Abstract The combination therapy of lumacaftor and ivacaftor (Orkambi®) is approved for patients bearing the major cystic fibrosis (CF) mutation: ΔF508. It has been predicted that Orkambi® could treat patients with rarer mutations of similar “theratype”; however, a standardized approach confirming e...

Full description

Saved in:
Bibliographic Details
Main Authors: Steven V Molinski, Saumel Ahmadi, Wan Ip, Hong Ouyang, Adriana Villella, John P Miller, Po‐Shun Lee, Kethika Kulleperuma, Kai Du, Michelle Di Paola, Paul DW Eckford, Onofrio Laselva, Ling Jun Huan, Leigh Wellhauser, Ellen Li, Peter N Ray, Régis Pomès, Theo J Moraes, Tanja Gonska, Felix Ratjen, Christine E Bear
Format: Article
Language:English
Published: Springer Nature 2017-06-01
Series:EMBO Molecular Medicine
Subjects:
amplifier
c.3700 A>G
CFTR
CRISPR/Cas9
cystic fibrosis
Online Access:https://doi.org/10.15252/emmm.201607137
Tags: Add Tag
No Tags, Be the first to tag this record!

Internet

https://doi.org/10.15252/emmm.201607137

Similar Items