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

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 effica...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2017
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5582412/
https://www.ncbi.nlm.nih.gov/pubmed/28667089
http://dx.doi.org/10.15252/emmm.201607137

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5582412/
https://www.ncbi.nlm.nih.gov/pubmed/28667089
http://dx.doi.org/10.15252/emmm.201607137

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