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Publications

Journal Article Review Epublish

PubMed: 42928365 DOI: 10.1038/s43856-026-01965-0

Theratyping for personalized medicine across cystic fibrosis disease models.

Commun Med (Lond) 6(1)

Authors: Valentino Bezzerri, Tarini Gunawardena, Tanja Gonska, Alvin P Chan, Rasha Salih, Francesca Lucca, Theo J Moraes, Steven D Freedman, Marco Cipolli

Affiliations

  • Cystic Fibrosis Center, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.
  • Department of Life Sciences, Health, and Health Professions, Link Campus University, Rome, Italy.
  • Program in Translational Medicine, Hospital for Sick Children, Toronto, ON, Canada.
  • Pediatric Gastroenterology, Toronto Hospital for Sick Children, Toronto, ON, Canada.
  • Pediatric Gastroenterology, David Geffen School of Medicine, University of California, Los Angeles, CA, USA.
  • Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON, Canada.
  • Department of Pediatrics, Division of Respiratory Medicine, Hospital for Sick Children, Toronto, ON, Canada.
  • Beth Israel Deaconess, Medical Center Harvard Medical School, Boston, MA, USA.
  • Cystic Fibrosis Center, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.

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.

MeSH

Fibrosis, Cystic Fibrosis, Precision Medicine, Research, Therapeutics