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

ABSTRACT: 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 requires correlation of in-vitro responses to in-vivo measures of clinical biomarkers of disease outcomes. While initial efforts have shown some success, this translation requires methodologies that are sensitive enough to capture treatment responses in a CF population that now predominantly has mild lung disease. Future development of in-vitro and in-vivo biomarkers will facilitat

SUBMITTER: Dumas MP 

PROVIDER: S-EPMC8577330 | biostudies-literature | 2021 Nov

REPOSITORIES: biostudies-literature