Human Pluripotent Stem Cells and Organoids: Current Trends and Future Perspectives

A special issue of Cells (ISSN 2073-4409). This special issue belongs to the section "Stem Cells".

Deadline for manuscript submissions: 1 June 2026 | Viewed by 631

Special Issue Editors


E-Mail Website
Guest Editor
Unité Mixte de Recherche (UMR_S) 1193, INSERM/Université Paris-Saclay, F-94800 Villejuif, France
Interests: hiPSCs; organoids; liver

E-Mail Website
Guest Editor
Unité Mixte de Recherche (UMR_S) 1193, INSERM/Université Paris-Saclay, F-94800 Villejuif, France
Interests: hiPSCs; organoids; liver

Special Issue Information

Dear Colleagues,

Human pluripotent stem cells (hPSCs), including embryonic stem cells and induced pluripotent stem cells, have become indispensable tools for advancing developmental biology, disease modeling, and regenerative medicine. The emergence of organoid technologies derived from hPSCs has provided unprecedented opportunities to recapitulate aspects of human tissue architecture and function in vitro, offering a powerful alternative to traditional two-dimensional cultures and animal models.

This rapidly evolving field is now at the crossroads of several disciplines. Refining protocols to enhance reproducibility, reduce heterogeneity, and achieve long-term maturation remains a critical challenge. Moreover, recent efforts integrating genome editing, biomaterials engineering, microfluidics, vascularization strategies, and immune system components are expanding the scope of organoid applications. Such advances are paving the way for precision medicine approaches, high-throughput drug discovery, and potentially clinical translation.

This Special Issue of Cells, “Human Pluripotent Stem Cells and Organoids: Current Trends and Future Perspectives”, welcomes original research articles and reviews that address recent developments across this spectrum. Topics of interest include novel culture methods, single-cell and multi-omics profiling, disease-specific models, therapeutic applications, and bioengineering innovations. By gathering contributions from leading experts, this issue aims to provide an integrated perspective on current achievements and future directions for hPSC and organoid research.

Dr. Eléanor Luce
Dr. Anne Dubart-Kupperschmitt
Guest Editors

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Keywords

  • human pluripotent stem cells
  • organoids
  • disease modeling
  • regenerative medicine
  • genome editing
  • single-cell omics
  • tissue engineering
  • drug discovery
  • precision medicine

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Published Papers (1 paper)

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Research

19 pages, 5107 KB  
Article
CFTR Modulator Response in Nasal Organoids Derived from People with Cystic Fibrosis
by Stefania Lo Cicero, Germana Castelli, Aurora Ceci, Anna Maria Cerio, Giovanna Blaconà, Mariarita Virgulti, Sara Allushi, Giovanni Sette, Francesca Spadaro, Felice Amato, Paola Melotti, Claudio Sorio, Giuseppe Cimino, Mauro Biffoni, Marco Lucarelli and Adriana Eramo
Cells 2025, 14(23), 1914; https://doi.org/10.3390/cells14231914 - 2 Dec 2025
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Abstract
Despite the progressive extension of CFTR variant eligibility to the triple combination of elexacaftor/tezacaftor/ivacaftor (ETI), most rare CFTR pathogenic variants remain ineligible for CFTR modulators. It is crucial to determine whether unexplored variants are rescuable by clinical modulators and to identify innovative therapeutic [...] Read more.
Despite the progressive extension of CFTR variant eligibility to the triple combination of elexacaftor/tezacaftor/ivacaftor (ETI), most rare CFTR pathogenic variants remain ineligible for CFTR modulators. It is crucial to determine whether unexplored variants are rescuable by clinical modulators and to identify innovative therapeutic strategies for rescuing non-responder variants. The approach known as “theratyping” (in vitro testing of genotypes) has been accepted by the Food and Drug Administration (FDA) for the extension of clinical modulators’ approval for in vitro responding genotypes. We used one of the most advanced models for theratyping: organoids derived from nasal epithelia of people with cystic fibrosis (pwCF). We optimized the forskolin-induced swelling (FIS) of organoids to assess CFTR basal or modulator-restored function. Nasal organoids mimicked the original epithelial tissue, CFTR residual activity, and modulator response. We set up the FIS assay using nasal organoids with reference genotypes and theratyped 38 rare (non-F508del) CFTR genotypes, either eligible or non-eligible for FDA approval, for treatment with ETI or ivacaftor. We found strong correspondence between the in vitro response of CFTR variants to modulators and their FDA approval status. Additionally, some previously uncharacterized CFTR variants have proven responsive to clinical modulators, with significant therapeutic implications. These results suggest that the nasal organoid FIS assay, pending confirmation of the prediction in the corresponding pwCF, might be considered as a powerful in vitro tool to predict modulator efficacy in each pwCF, guiding out-of-label prescription in CF, and to identify uncharacterized variants responsive to modulators. This approach may allow comparison of the efficacy of different therapeutics or the identification of innovative strategies for non-responding genotypes, improving personalized therapy and quality of life for pwCF. Full article
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