Theratyping for personalized medicine across cystic fibrosis disease models

Abstract

In the era of CFTR modulators, cystic fibrosis (CF) is being profoundly reshaped for treatment-eligible individuals. Theratyping, an ex vivo strategy assessing individual responses to CFTR modulators, supports precision medicine, particularly for rare variants where conventional trials are limited. In this Review, we discussed advances in CF disease modelling, highlighting strengths, limitations and translational relevance. We also outlined future directions aligned with the Replacement, Reduction, and Refinement (3 R) ethical principles in animal research, including organ-on-a-chip technologies, to improve mechanistic insight and preclinical predictivity. CF research offers therefore a paradigmatic framework for therapeutic innovation in other rare genetic diseases.

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BibTeX RIS

BibTeX

@article{bezzerri2026theratyping,
  title = {{Theratyping for personalized medicine across cystic fibrosis disease models}},
  author = {Valentino Bezzerri and Tarini Gunawardena and Tanja Gonska and Alvin P. Chan and Rasha Salih and Francesca Lucca and Theo J. Moraes and Steven D. Freedman and Marco Cipolli},
  journal = {Communications Medicine},
  year = {2026},
  month = oct,
  doi = {10.1038/s43856-026-01965-0},
  url = {https://www.nature.com/articles/s43856-026-01965-0},
  note = {CC BY-NC-ND 4.0},
}

RIS

TY  - JOUR
TI  - Theratyping for personalized medicine across cystic fibrosis disease models
AU  - Valentino Bezzerri
AU  - Tarini Gunawardena
AU  - Tanja Gonska
AU  - Alvin P. Chan
AU  - Rasha Salih
AU  - Francesca Lucca
AU  - Theo J. Moraes
AU  - Steven D. Freedman
AU  - Marco Cipolli
PY  - 2026
DA  - 2026/10/25
JO  - Communications Medicine
DO  - 10.1038/s43856-026-01965-0
UR  - https://www.nature.com/articles/s43856-026-01965-0
AB  - In the era of CFTR modulators, cystic fibrosis (CF) is being profoundly reshaped for treatment-eligible individuals. Theratyping, an ex vivo strategy assessing individual responses to CFTR modulators, supports precision medicine, particularly for rare variants where conventional trials are limited. In this Review, we discussed advances in CF disease modelling, highlighting strengths, limitations and translational relevance. We also outlined future directions aligned with the Replacement, Reduction, and Refinement (3 R) ethical principles in animal research, including organ-on-a-chip technologies, to improve mechanistic insight and preclinical predictivity. CF research offers therefore a paradigmatic framework for therapeutic innovation in other rare genetic diseases.
SN  - 2730-664X
ER  -

Abstract

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