Massively parallel reporter assays and mouse transgenic assays provide correlated and complementary information about neuronal enhancer activity

Abstract High-throughput massively parallel reporter assays (MPRAs) and phenotype-rich in vivo transgenic mouse assays are two potentially complementary ways to study the impact of noncoding variants associated with psychiatric diseases. Here, we investigate the utility of combining these assays. Sp...

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Main Authors: Michael Kosicki, Dianne Laboy Cintrón, Pia Keukeleire, Max Schubach, Nicholas F. Page, Ilias Georgakopoulos-Soares, Jennifer A. Akiyama, Ingrid Plajzer-Frick, Catherine S. Novak, Momoe Kato, Riana D. Hunter, Kianna von Maydell, Sarah Barton, Patrick Godfrey, Erik Beckman, Stephan J. Sanders, Martin Kircher, Len A. Pennacchio, Nadav Ahituv
Format: Article
Language:English
Published: Nature Portfolio 2025-05-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-60064-1
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author Michael Kosicki
Dianne Laboy Cintrón
Pia Keukeleire
Max Schubach
Nicholas F. Page
Ilias Georgakopoulos-Soares
Jennifer A. Akiyama
Ingrid Plajzer-Frick
Catherine S. Novak
Momoe Kato
Riana D. Hunter
Kianna von Maydell
Sarah Barton
Patrick Godfrey
Erik Beckman
Stephan J. Sanders
Martin Kircher
Len A. Pennacchio
Nadav Ahituv
author_facet Michael Kosicki
Dianne Laboy Cintrón
Pia Keukeleire
Max Schubach
Nicholas F. Page
Ilias Georgakopoulos-Soares
Jennifer A. Akiyama
Ingrid Plajzer-Frick
Catherine S. Novak
Momoe Kato
Riana D. Hunter
Kianna von Maydell
Sarah Barton
Patrick Godfrey
Erik Beckman
Stephan J. Sanders
Martin Kircher
Len A. Pennacchio
Nadav Ahituv
author_sort Michael Kosicki
collection DOAJ
description Abstract High-throughput massively parallel reporter assays (MPRAs) and phenotype-rich in vivo transgenic mouse assays are two potentially complementary ways to study the impact of noncoding variants associated with psychiatric diseases. Here, we investigate the utility of combining these assays. Specifically, we carry out an MPRA in induced human neurons on over 50,000 sequences derived from fetal neuronal ATAC-seq datasets and enhancers validated in mouse assays. We also test the impact of over 20,000 variants, including synthetic mutations and 167 common variants associated with psychiatric disorders. We find a strong and specific correlation between MPRA and mouse neuronal enhancer activity. Four out of five tested variants with significant MPRA effects affected neuronal enhancer activity in mouse embryos. Mouse assays also reveal pleiotropic variant effects that could not be observed in MPRA. Our work provides a catalog of functional neuronal enhancers and variant effects and highlights the effectiveness of combining MPRAs and mouse transgenic assays.
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spelling doaj-art-8e3ee87d4e7a417fb6beb3ca59926f462025-08-20T01:53:23ZengNature PortfolioNature Communications2041-17232025-05-0116111310.1038/s41467-025-60064-1Massively parallel reporter assays and mouse transgenic assays provide correlated and complementary information about neuronal enhancer activityMichael Kosicki0Dianne Laboy Cintrón1Pia Keukeleire2Max Schubach3Nicholas F. Page4Ilias Georgakopoulos-Soares5Jennifer A. Akiyama6Ingrid Plajzer-Frick7Catherine S. Novak8Momoe Kato9Riana D. Hunter10Kianna von Maydell11Sarah Barton12Patrick Godfrey13Erik Beckman14Stephan J. Sanders15Martin Kircher16Len A. Pennacchio17Nadav Ahituv18Environmental Genomics & Systems Biology Division, Lawrence Berkeley National LaboratoryDepartment of Bioengineering and Therapeutic Sciences, University of California San FranciscoInstitute of Human Genetics, University Medical Center Schleswig-Holstein, University of LübeckBerlin Institute of Health at Charité – Universitätsmedizin BerlinDepartment of Bioengineering and Therapeutic Sciences, University of California San FranciscoInstitute for Personalized Medicine, Department of Biochemistry and Molecular Biology, The Pennsylvania State University College of MedicineEnvironmental Genomics & Systems Biology Division, Lawrence Berkeley National LaboratoryEnvironmental Genomics & Systems Biology Division, Lawrence Berkeley National LaboratoryEnvironmental Genomics & Systems Biology Division, Lawrence Berkeley National LaboratoryEnvironmental Genomics & Systems Biology Division, Lawrence Berkeley National LaboratoryEnvironmental Genomics & Systems Biology Division, Lawrence Berkeley National LaboratoryEnvironmental Genomics & Systems Biology Division, Lawrence Berkeley National LaboratoryEnvironmental Genomics & Systems Biology Division, Lawrence Berkeley National LaboratoryEnvironmental Genomics & Systems Biology Division, Lawrence Berkeley National LaboratoryEnvironmental Genomics & Systems Biology Division, Lawrence Berkeley National LaboratoryInstitute for Human Genetics, University of California San FranciscoInstitute of Human Genetics, University Medical Center Schleswig-Holstein, University of LübeckEnvironmental Genomics & Systems Biology Division, Lawrence Berkeley National LaboratoryDepartment of Bioengineering and Therapeutic Sciences, University of California San FranciscoAbstract High-throughput massively parallel reporter assays (MPRAs) and phenotype-rich in vivo transgenic mouse assays are two potentially complementary ways to study the impact of noncoding variants associated with psychiatric diseases. Here, we investigate the utility of combining these assays. Specifically, we carry out an MPRA in induced human neurons on over 50,000 sequences derived from fetal neuronal ATAC-seq datasets and enhancers validated in mouse assays. We also test the impact of over 20,000 variants, including synthetic mutations and 167 common variants associated with psychiatric disorders. We find a strong and specific correlation between MPRA and mouse neuronal enhancer activity. Four out of five tested variants with significant MPRA effects affected neuronal enhancer activity in mouse embryos. Mouse assays also reveal pleiotropic variant effects that could not be observed in MPRA. Our work provides a catalog of functional neuronal enhancers and variant effects and highlights the effectiveness of combining MPRAs and mouse transgenic assays.https://doi.org/10.1038/s41467-025-60064-1
spellingShingle Michael Kosicki
Dianne Laboy Cintrón
Pia Keukeleire
Max Schubach
Nicholas F. Page
Ilias Georgakopoulos-Soares
Jennifer A. Akiyama
Ingrid Plajzer-Frick
Catherine S. Novak
Momoe Kato
Riana D. Hunter
Kianna von Maydell
Sarah Barton
Patrick Godfrey
Erik Beckman
Stephan J. Sanders
Martin Kircher
Len A. Pennacchio
Nadav Ahituv
Massively parallel reporter assays and mouse transgenic assays provide correlated and complementary information about neuronal enhancer activity
Nature Communications
title Massively parallel reporter assays and mouse transgenic assays provide correlated and complementary information about neuronal enhancer activity
title_full Massively parallel reporter assays and mouse transgenic assays provide correlated and complementary information about neuronal enhancer activity
title_fullStr Massively parallel reporter assays and mouse transgenic assays provide correlated and complementary information about neuronal enhancer activity
title_full_unstemmed Massively parallel reporter assays and mouse transgenic assays provide correlated and complementary information about neuronal enhancer activity
title_short Massively parallel reporter assays and mouse transgenic assays provide correlated and complementary information about neuronal enhancer activity
title_sort massively parallel reporter assays and mouse transgenic assays provide correlated and complementary information about neuronal enhancer activity
url https://doi.org/10.1038/s41467-025-60064-1
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