A differential requirement for ciliary transition zone proteins in human and mouse neural progenitor fate specification

Abstract Studying ciliary genes in the context of the human central nervous system is crucial for understanding the underlying causes of neurodevelopmental ciliopathies. Here, we use pluripotent stem cell-derived spinal organoids to reveal distinct functions of the ciliopathy gene RPGRIP1L in humans...

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Main Authors: Antonia Wiegering, Isabelle Anselme, Ludovica Brunetti, Laura Metayer-Derout, Damelys Calderon, Sophie Thomas, Stéphane Nedelec, Alexis Eschstruth, Valentina Serpieri, Martin Catala, Christophe Antoniewski, Sylvie Schneider-Maunoury, Aline Stedman
Format: Article
Language:English
Published: Nature Portfolio 2025-04-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-58554-3
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author Antonia Wiegering
Isabelle Anselme
Ludovica Brunetti
Laura Metayer-Derout
Damelys Calderon
Sophie Thomas
Stéphane Nedelec
Alexis Eschstruth
Valentina Serpieri
Martin Catala
Christophe Antoniewski
Sylvie Schneider-Maunoury
Aline Stedman
author_facet Antonia Wiegering
Isabelle Anselme
Ludovica Brunetti
Laura Metayer-Derout
Damelys Calderon
Sophie Thomas
Stéphane Nedelec
Alexis Eschstruth
Valentina Serpieri
Martin Catala
Christophe Antoniewski
Sylvie Schneider-Maunoury
Aline Stedman
author_sort Antonia Wiegering
collection DOAJ
description Abstract Studying ciliary genes in the context of the human central nervous system is crucial for understanding the underlying causes of neurodevelopmental ciliopathies. Here, we use pluripotent stem cell-derived spinal organoids to reveal distinct functions of the ciliopathy gene RPGRIP1L in humans and mice, and uncover an unexplored role for cilia in human axial patterning. Previous research has emphasized Rpgrip1l critical functions in mouse brain and spinal cord development through the regulation of SHH/GLI pathway. Here, we show that RPGRIP1L is not required for SHH activation or motoneuron lineage commitment in human spinal progenitors and that this feature is shared by another ciliopathy gene, TMEM67. Furthermore, human RPGRIP1L-mutant motoneurons adopt hindbrain and cervical identities instead of caudal brachial identity. Temporal transcriptome analysis reveals that this antero-posterior patterning defect originates in early axial progenitors and correlates with cilia loss. These findings provide important insights into the role of cilia in human neural development.
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spelling doaj-art-ecb54a5184714fd1b89c480aa2a26e022025-08-20T03:07:43ZengNature PortfolioNature Communications2041-17232025-04-0116111910.1038/s41467-025-58554-3A differential requirement for ciliary transition zone proteins in human and mouse neural progenitor fate specificationAntonia Wiegering0Isabelle Anselme1Ludovica Brunetti2Laura Metayer-Derout3Damelys Calderon4Sophie Thomas5Stéphane Nedelec6Alexis Eschstruth7Valentina Serpieri8Martin Catala9Christophe Antoniewski10Sylvie Schneider-Maunoury11Aline Stedman12Sorbonne Université, CNRS, Inserm, Development, Adaptation and Aging, Dev2ASorbonne Université, CNRS, Inserm, Development, Adaptation and Aging, Dev2ASorbonne Université, CNRS, Inserm, Development, Adaptation and Aging, Dev2ASorbonne Université, CNRS, Inserm, Development, Adaptation and Aging, Dev2AINSERM UMR 1163, Institut Imagine, Université Paris CitéINSERM UMR 1163, Institut Imagine, Université Paris CitéSorbonne Université, Inserm, Institut du Fer à Moulin, UMR-S 1270Sorbonne Université, CNRS, Inserm, Development, Adaptation and Aging, Dev2ADepartment of Molecular Medicine, University of PaviaSorbonne Université, CNRS, Inserm, Development, Adaptation and Aging, Dev2ASorbonne Université, CNRS, Inserm, Institut de Biologie Paris SeineSorbonne Université, CNRS, Inserm, Development, Adaptation and Aging, Dev2ASorbonne Université, CNRS, Inserm, Development, Adaptation and Aging, Dev2AAbstract Studying ciliary genes in the context of the human central nervous system is crucial for understanding the underlying causes of neurodevelopmental ciliopathies. Here, we use pluripotent stem cell-derived spinal organoids to reveal distinct functions of the ciliopathy gene RPGRIP1L in humans and mice, and uncover an unexplored role for cilia in human axial patterning. Previous research has emphasized Rpgrip1l critical functions in mouse brain and spinal cord development through the regulation of SHH/GLI pathway. Here, we show that RPGRIP1L is not required for SHH activation or motoneuron lineage commitment in human spinal progenitors and that this feature is shared by another ciliopathy gene, TMEM67. Furthermore, human RPGRIP1L-mutant motoneurons adopt hindbrain and cervical identities instead of caudal brachial identity. Temporal transcriptome analysis reveals that this antero-posterior patterning defect originates in early axial progenitors and correlates with cilia loss. These findings provide important insights into the role of cilia in human neural development.https://doi.org/10.1038/s41467-025-58554-3
spellingShingle Antonia Wiegering
Isabelle Anselme
Ludovica Brunetti
Laura Metayer-Derout
Damelys Calderon
Sophie Thomas
Stéphane Nedelec
Alexis Eschstruth
Valentina Serpieri
Martin Catala
Christophe Antoniewski
Sylvie Schneider-Maunoury
Aline Stedman
A differential requirement for ciliary transition zone proteins in human and mouse neural progenitor fate specification
Nature Communications
title A differential requirement for ciliary transition zone proteins in human and mouse neural progenitor fate specification
title_full A differential requirement for ciliary transition zone proteins in human and mouse neural progenitor fate specification
title_fullStr A differential requirement for ciliary transition zone proteins in human and mouse neural progenitor fate specification
title_full_unstemmed A differential requirement for ciliary transition zone proteins in human and mouse neural progenitor fate specification
title_short A differential requirement for ciliary transition zone proteins in human and mouse neural progenitor fate specification
title_sort differential requirement for ciliary transition zone proteins in human and mouse neural progenitor fate specification
url https://doi.org/10.1038/s41467-025-58554-3
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