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Article

Perspectives on the translation of in-vitro studies to precision medicine in Cystic Fibrosis.

EBioMedicine - 1 Nov 2021

Dumas Marie-Pier, Xia Sunny, Bear Christine E, Ratjen Felix

Abstract excerpt

Recent strides towards precision medicine in Cystic Fibrosis (CF) have been made possible by patient-derived in-vitro assays with the potential to predict clinical response to small molecule-based therapies. Here, we discuss the status of primary and stem-cell derived tissues used to evaluate the preclinical efficacy of CFTR modulators highlighting both their potential and limitations. Validation of these assays...

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Identifiers

PMID: 34740114
DOI: 10.1016/j.ebiom.2021.103660
PMCID: PMC8577330

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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