The CF Canada-Sick Kids Program in individual CF therapy: A resource for the advancement of personalized medicine in CF.

Paul D W Eckford, Jacqueline McCormack, Lise Munsie,Gengming He,Sanja Stanojevic,Sergio L Pereira,Karen Ho,Julie Avolio,Claire Bartlett,Jin Ye Yang,Amy P Wong,Leigh Wellhauser,Ling Jun Huan, Jia Xin Jiang,Hong Ouyang,Kai Du,Michelle Klingel,Lianna Kyriakopoulou,Tanja Gonska,Theo J Moraes,Lisa J Strug,Janet Rossant,Felix Ratjen,Christine E Bear

Journal of Cystic Fibrosis（2019）

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摘要

This accessible resource will enable development of tools that predict individual outcomes to current and emerging modulators targeting F508del-CFTR and facilitate therapy discovery for rare CF causing mutations.

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关键词

Precision medicine,Patient-specific primary tissues,Nasal,Cultures,Drug responses,Pluripotent stem cells,Resource for discovery,Whole genome sequencing,Clinical outcome data,RNAseq

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