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Article

Personalized Medicine Based on Nasal Epithelial Cells: Comparative Studies with Rectal Biopsies and Intestinal Organoids

BioISI—Biosystems and Integrative Sciences Institute, Faculty of Sciences, University of Lisboa, 1749-016 Lisboa, Portugal
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Academic Editors: Cornelis K. van der Ent and Harry G. M. Heijerman
J. Pers. Med. 2021, 11(5), 421; https://doi.org/10.3390/jpm11050421
Received: 24 April 2021 / Revised: 9 May 2021 / Accepted: 12 May 2021 / Published: 16 May 2021
(This article belongs to the Special Issue Cystic Fibrosis and Personalized Medicine)
As highly effective CFTR modulator therapies (HEMT) emerge, there is an unmet need to find effective drugs for people with CF (PwCF) with ultra-rare mutations who are too few for classical clinical trials and for whom there are no drug discovery programs. Therefore, biomarkers reliably predicting the benefit from CFTR modulator therapies are essential to find effective drugs for PwCF through personalized approaches termed theranostics. Here, we assess CFTR basal function and the individual responses to CFTR modulators in primary human nasal epithelial (pHNE) cells from PwCF carrying rare mutations and compare these measurements with those in native rectal biopsies and intestinal organoids, respectively, in the same individual. The basal function in pHNEs shows good correlation with CFTR basal function in rectal biopsies. In parallel, CFTR rescue in pHNEs by CFTR modulators correlates to that in intestinal organoids. Altogether, results show that pHNEs are a bona fide theranostic model to assess CFTR rescue by CFTR modulator drugs, in particular for PwCF and rare mutations. View Full-Text
Keywords: CFTR; human nasal epithelial cells; rectal biopsies; intestinal organoids; CFTR modulators; theranostic CFTR; human nasal epithelial cells; rectal biopsies; intestinal organoids; CFTR modulators; theranostic
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MDPI and ACS Style

Silva, I.A.L.; Railean, V.; Duarte, A.; Amaral, M.D. Personalized Medicine Based on Nasal Epithelial Cells: Comparative Studies with Rectal Biopsies and Intestinal Organoids. J. Pers. Med. 2021, 11, 421. https://doi.org/10.3390/jpm11050421

AMA Style

Silva IAL, Railean V, Duarte A, Amaral MD. Personalized Medicine Based on Nasal Epithelial Cells: Comparative Studies with Rectal Biopsies and Intestinal Organoids. Journal of Personalized Medicine. 2021; 11(5):421. https://doi.org/10.3390/jpm11050421

Chicago/Turabian Style

Silva, Iris A.L., Violeta Railean, Aires Duarte, and Margarida D. Amaral 2021. "Personalized Medicine Based on Nasal Epithelial Cells: Comparative Studies with Rectal Biopsies and Intestinal Organoids" Journal of Personalized Medicine 11, no. 5: 421. https://doi.org/10.3390/jpm11050421

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