Single-cell analysis of dup15q syndrome reveals developmental and postnatal molecular changes in autism

Abstract Duplication 15q (dup15q) syndrome is a leading genetic cause of autism spectrum disorder, offering a key model for studying autism-related mechanisms. Using single-cell and single-nucleus RNA sequencing of cortical organoids from dup15q patient-derived iPSCs and post-mortem brain samples, w...

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Main Authors: Yonatan Perez, Dmitry Velmeshev, Li Wang, Matthew L. White, Clara Siebert, Jennifer Baltazar, Guolong Zuo, Juan Andrés Moriano, Songcang Chen, David M. Steffen, Natalia Garcia Dutton, Shaohui Wang, Brittney Wick, Maximilian Haeussler, Stormy Chamberlain, Arturo Alvarez-Buylla, Arnold Kriegstein
Format: Article
Language:English
Published: Nature Portfolio 2025-07-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-61184-4
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author Yonatan Perez
Dmitry Velmeshev
Li Wang
Matthew L. White
Clara Siebert
Jennifer Baltazar
Guolong Zuo
Juan Andrés Moriano
Songcang Chen
David M. Steffen
Natalia Garcia Dutton
Shaohui Wang
Brittney Wick
Maximilian Haeussler
Stormy Chamberlain
Arturo Alvarez-Buylla
Arnold Kriegstein
author_facet Yonatan Perez
Dmitry Velmeshev
Li Wang
Matthew L. White
Clara Siebert
Jennifer Baltazar
Guolong Zuo
Juan Andrés Moriano
Songcang Chen
David M. Steffen
Natalia Garcia Dutton
Shaohui Wang
Brittney Wick
Maximilian Haeussler
Stormy Chamberlain
Arturo Alvarez-Buylla
Arnold Kriegstein
author_sort Yonatan Perez
collection DOAJ
description Abstract Duplication 15q (dup15q) syndrome is a leading genetic cause of autism spectrum disorder, offering a key model for studying autism-related mechanisms. Using single-cell and single-nucleus RNA sequencing of cortical organoids from dup15q patient-derived iPSCs and post-mortem brain samples, we identify increased glycolysis, disrupted layer-specific marker expression, and aberrant morphology in deep-layer neurons during fetal-stage organoid development. In adolescent-adult postmortem brains, upper-layer neurons exhibit heightened transcriptional burden related to synaptic signaling, a pattern shared with idiopathic autism. Using spatial transcriptomics, we confirm these cell-type-specific disruptions in brain tissue. By gene co-expression network analysis, we reveal disease-associated modules that are well preserved between postmortem and organoid samples, suggesting metabolic dysregulation that may lead to altered neuron projection, synaptic dysfunction, and neuron hyperexcitability in dup15q syndrome.
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spelling doaj-art-a3b3a586454341ce8a22152004ea2d382025-08-20T03:37:37ZengNature PortfolioNature Communications2041-17232025-07-0116111610.1038/s41467-025-61184-4Single-cell analysis of dup15q syndrome reveals developmental and postnatal molecular changes in autismYonatan Perez0Dmitry Velmeshev1Li Wang2Matthew L. White3Clara Siebert4Jennifer Baltazar5Guolong Zuo6Juan Andrés Moriano7Songcang Chen8David M. Steffen9Natalia Garcia Dutton10Shaohui Wang11Brittney Wick12Maximilian Haeussler13Stormy Chamberlain14Arturo Alvarez-Buylla15Arnold Kriegstein16Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoGenomics Institute, University of CaliforniaGenomics Institute, University of CaliforniaDepartments of Genetics and Genome Sciences and Pediatrics, Connecticut Children’s Medical Center, University of Connecticut Health Center, 400 Farmington AvenueEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoEli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California, San FranciscoAbstract Duplication 15q (dup15q) syndrome is a leading genetic cause of autism spectrum disorder, offering a key model for studying autism-related mechanisms. Using single-cell and single-nucleus RNA sequencing of cortical organoids from dup15q patient-derived iPSCs and post-mortem brain samples, we identify increased glycolysis, disrupted layer-specific marker expression, and aberrant morphology in deep-layer neurons during fetal-stage organoid development. In adolescent-adult postmortem brains, upper-layer neurons exhibit heightened transcriptional burden related to synaptic signaling, a pattern shared with idiopathic autism. Using spatial transcriptomics, we confirm these cell-type-specific disruptions in brain tissue. By gene co-expression network analysis, we reveal disease-associated modules that are well preserved between postmortem and organoid samples, suggesting metabolic dysregulation that may lead to altered neuron projection, synaptic dysfunction, and neuron hyperexcitability in dup15q syndrome.https://doi.org/10.1038/s41467-025-61184-4
spellingShingle Yonatan Perez
Dmitry Velmeshev
Li Wang
Matthew L. White
Clara Siebert
Jennifer Baltazar
Guolong Zuo
Juan Andrés Moriano
Songcang Chen
David M. Steffen
Natalia Garcia Dutton
Shaohui Wang
Brittney Wick
Maximilian Haeussler
Stormy Chamberlain
Arturo Alvarez-Buylla
Arnold Kriegstein
Single-cell analysis of dup15q syndrome reveals developmental and postnatal molecular changes in autism
Nature Communications
title Single-cell analysis of dup15q syndrome reveals developmental and postnatal molecular changes in autism
title_full Single-cell analysis of dup15q syndrome reveals developmental and postnatal molecular changes in autism
title_fullStr Single-cell analysis of dup15q syndrome reveals developmental and postnatal molecular changes in autism
title_full_unstemmed Single-cell analysis of dup15q syndrome reveals developmental and postnatal molecular changes in autism
title_short Single-cell analysis of dup15q syndrome reveals developmental and postnatal molecular changes in autism
title_sort single cell analysis of dup15q syndrome reveals developmental and postnatal molecular changes in autism
url https://doi.org/10.1038/s41467-025-61184-4
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