BIBLIOS

Document type
Journal articles

Document subtype
Full paper

Title
Advances in Preclinical In Vitro Models for the Translation of Precision Medicine for Cystic Fibrosis

Participants in the publication
Iris A. L. Silva (Author)
Onofrio Laselva (Author)
Miquéias Lopes-Pacheco (Author)
BioISI

Summary
The development of preclinical in vitro models has provided significant progress to the\nstudies of cystic fibrosis (CF), a frequently fatal monogenic disease caused by mutations in the gene\nencoding the CF transmembrane conductance regulator (CFTR) protein. Numerous cell lines were\ngenerated over the last 30 years and they have been instrumental not only in enhancing the understanding\nof CF pathological mechanisms but also in developing therapies targeting the underlying\ndefects in CFTR mutations with further validation in patient-derived samples. Furthermore, recent\nadvances toward precision medicine in CF have been made possible by optimizing protocols and\nestablishing novel assays using human bronchial, nasal and rectal tissues, and by progressing from\ntwo-dimensional monocultures to more complex three-dimensional culture platforms. These models\nalso enable to potentially predict clinical efficacy and responsiveness to CFTR modulator therapies\nat an individual level. In parallel, advanced systems, such as induced pluripotent stem cells and\norgan-on-a-chip, continue to be developed in order to more closely recapitulate human physiology\nfor disease modeling and drug testing. In this review, we have highlighted novel and optimized cell\nmodels that are being used in CF research to develop novel CFTR-directed therapies (or alternative\ntherapeutic interventions) and to expand the usage of existing modulator drugs to common and rare\nCF-causing mutations.


Where published
Journal of Personalized Medicine

Publication Identifiers
ISSN - 2075-4426

Publisher
MDPI AG



Document Identifiers
DOI - https://doi.org/10.3390/jpm12081321
URL - http://dx.doi.org/10.3390/jpm12081321

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