Human iPSC-derived neural stem cells displaying radial glia signature exhibit long-term safety in mice

Abstract Human induced pluripotent stem cell-derived neural stem/progenitor cells (hiPSC-NSCs) hold promise for treating neurodegenerative and demyelinating disorders. However, comprehensive studies on their identity and safety remain limited. In this study, we demonstrate that hiPSC-NSCs adopt a ra...

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Main Authors: Marco Luciani, Chiara Garsia, Stefano Beretta, Ingrid Cifola, Clelia Peano, Ivan Merelli, Luca Petiti, Annarita Miccio, Vasco Meneghini, Angela Gritti
Format: Article
Language:English
Published: Nature Portfolio 2024-11-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-024-53613-7
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author Marco Luciani
Chiara Garsia
Stefano Beretta
Ingrid Cifola
Clelia Peano
Ivan Merelli
Luca Petiti
Annarita Miccio
Vasco Meneghini
Angela Gritti
author_facet Marco Luciani
Chiara Garsia
Stefano Beretta
Ingrid Cifola
Clelia Peano
Ivan Merelli
Luca Petiti
Annarita Miccio
Vasco Meneghini
Angela Gritti
author_sort Marco Luciani
collection DOAJ
description Abstract Human induced pluripotent stem cell-derived neural stem/progenitor cells (hiPSC-NSCs) hold promise for treating neurodegenerative and demyelinating disorders. However, comprehensive studies on their identity and safety remain limited. In this study, we demonstrate that hiPSC-NSCs adopt a radial glia-associated signature, sharing key epigenetic and transcriptional characteristics with human fetal neural stem cells (hfNSCs) while exhibiting divergent profiles from glioblastoma stem cells. Long-term transplantation studies in mice showed robust and stable engraftment of hiPSC-NSCs, with predominant differentiation into glial cells and no evidence of tumor formation. Additionally, we identified the Sterol Regulatory Element Binding Transcription Factor 1 (SREBF1) as a regulator of astroglial differentiation in hiPSC-NSCs. These findings provide valuable transcriptional and epigenetic reference datasets to prospectively define the maturation stage of NSCs derived from different hiPSC sources and demonstrate the long-term safety of hiPSC-NSCs, reinforcing their potential as a viable alternative to hfNSCs for clinical applications.
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publisher Nature Portfolio
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spelling doaj-art-8daf1e448b9c4dbdbc60072df517206a2025-08-20T02:18:31ZengNature PortfolioNature Communications2041-17232024-11-0115112410.1038/s41467-024-53613-7Human iPSC-derived neural stem cells displaying radial glia signature exhibit long-term safety in miceMarco Luciani0Chiara Garsia1Stefano Beretta2Ingrid Cifola3Clelia Peano4Ivan Merelli5Luca Petiti6Annarita Miccio7Vasco Meneghini8Angela Gritti9San Raffaele Telethon Institute for Gene Therapy, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Scientific InstituteSan Raffaele Telethon Institute for Gene Therapy, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Scientific InstituteSan Raffaele Telethon Institute for Gene Therapy, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Scientific InstituteInstitute for Biomedical Technologies (ITB), National Research Council (CNR), via F.lli Cervi 93, 20054 SegrateInstitute of Genetics and Biomedical Research, UoS of Milan, National Research Council, RozzanoSan Raffaele Telethon Institute for Gene Therapy, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Scientific InstituteInstitute for Biomedical Technologies (ITB), National Research Council (CNR), via F.lli Cervi 93, 20054 SegrateIMAGINE Institute, Université de Paris, Sorbonne Paris CitéSan Raffaele Telethon Institute for Gene Therapy, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Scientific InstituteSan Raffaele Telethon Institute for Gene Therapy, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Scientific InstituteAbstract Human induced pluripotent stem cell-derived neural stem/progenitor cells (hiPSC-NSCs) hold promise for treating neurodegenerative and demyelinating disorders. However, comprehensive studies on their identity and safety remain limited. In this study, we demonstrate that hiPSC-NSCs adopt a radial glia-associated signature, sharing key epigenetic and transcriptional characteristics with human fetal neural stem cells (hfNSCs) while exhibiting divergent profiles from glioblastoma stem cells. Long-term transplantation studies in mice showed robust and stable engraftment of hiPSC-NSCs, with predominant differentiation into glial cells and no evidence of tumor formation. Additionally, we identified the Sterol Regulatory Element Binding Transcription Factor 1 (SREBF1) as a regulator of astroglial differentiation in hiPSC-NSCs. These findings provide valuable transcriptional and epigenetic reference datasets to prospectively define the maturation stage of NSCs derived from different hiPSC sources and demonstrate the long-term safety of hiPSC-NSCs, reinforcing their potential as a viable alternative to hfNSCs for clinical applications.https://doi.org/10.1038/s41467-024-53613-7
spellingShingle Marco Luciani
Chiara Garsia
Stefano Beretta
Ingrid Cifola
Clelia Peano
Ivan Merelli
Luca Petiti
Annarita Miccio
Vasco Meneghini
Angela Gritti
Human iPSC-derived neural stem cells displaying radial glia signature exhibit long-term safety in mice
Nature Communications
title Human iPSC-derived neural stem cells displaying radial glia signature exhibit long-term safety in mice
title_full Human iPSC-derived neural stem cells displaying radial glia signature exhibit long-term safety in mice
title_fullStr Human iPSC-derived neural stem cells displaying radial glia signature exhibit long-term safety in mice
title_full_unstemmed Human iPSC-derived neural stem cells displaying radial glia signature exhibit long-term safety in mice
title_short Human iPSC-derived neural stem cells displaying radial glia signature exhibit long-term safety in mice
title_sort human ipsc derived neural stem cells displaying radial glia signature exhibit long term safety in mice
url https://doi.org/10.1038/s41467-024-53613-7
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