Mapping the genetic landscape establishing a tumor immune microenvironment favorable for anti-PD-1 response

Summary: Identifying host genetic factors modulating immune checkpoint inhibitor (ICI) efficacy is experimentally challenging. Our approach, utilizing the Collaborative Cross mouse genetic resource, fixes the tumor genomic configuration while varying host genetics. We find that response to anti-PD-1...

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Main Authors: Daniel A. Skelly, John P. Graham, Mingshan Cheng, Mayuko Furuta, Andrew Walter, Thomas A. Stoklasek, Hongyuan Yang, Timothy M. Stearns, Olivier Poirion, Ji-Gang Zhang, Jessica D.S. Grassmann, Diane Luo, William F. Flynn, Elise T. Courtois, Chih-Hao Chang, David V. Serreze, Francesca Menghi, Laura G. Reinholdt, Edison T. Liu
Format: Article
Language:English
Published: Elsevier 2025-05-01
Series:Cell Reports
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Online Access:http://www.sciencedirect.com/science/article/pii/S2211124725004693
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author Daniel A. Skelly
John P. Graham
Mingshan Cheng
Mayuko Furuta
Andrew Walter
Thomas A. Stoklasek
Hongyuan Yang
Timothy M. Stearns
Olivier Poirion
Ji-Gang Zhang
Jessica D.S. Grassmann
Diane Luo
William F. Flynn
Elise T. Courtois
Chih-Hao Chang
David V. Serreze
Francesca Menghi
Laura G. Reinholdt
Edison T. Liu
author_facet Daniel A. Skelly
John P. Graham
Mingshan Cheng
Mayuko Furuta
Andrew Walter
Thomas A. Stoklasek
Hongyuan Yang
Timothy M. Stearns
Olivier Poirion
Ji-Gang Zhang
Jessica D.S. Grassmann
Diane Luo
William F. Flynn
Elise T. Courtois
Chih-Hao Chang
David V. Serreze
Francesca Menghi
Laura G. Reinholdt
Edison T. Liu
author_sort Daniel A. Skelly
collection DOAJ
description Summary: Identifying host genetic factors modulating immune checkpoint inhibitor (ICI) efficacy is experimentally challenging. Our approach, utilizing the Collaborative Cross mouse genetic resource, fixes the tumor genomic configuration while varying host genetics. We find that response to anti-PD-1 (aPD1) immunotherapy is significantly heritable in four distinct murine tumor models (H2: 0.18–0.40). For the MC38 colorectal carcinoma system, we map four significant ICI response quantitative trait loci (QTLs) with significant epistatic interactions. The differentially expressed genes within these QTLs that define responder genetics are highly enriched for processes involving antigen processing and presentation, allograft rejection, and graft vs. host disease (all p < 1 × 10−10). Functional blockade of two top candidate immune targets, GM-CSF and IL-2RB, completely abrogates the MC38 transcriptional response to aPD1 therapy. Thus, our in vivo experimental platform is a powerful approach for discovery of host genetic factors that establish the tumor immune microenvironment propitious for ICI response.
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spelling doaj-art-d53d8f6672db4f648dba14e31189ee062025-08-20T02:15:20ZengElsevierCell Reports2211-12472025-05-0144511569810.1016/j.celrep.2025.115698Mapping the genetic landscape establishing a tumor immune microenvironment favorable for anti-PD-1 responseDaniel A. Skelly0John P. Graham1Mingshan Cheng2Mayuko Furuta3Andrew Walter4Thomas A. Stoklasek5Hongyuan Yang6Timothy M. Stearns7Olivier Poirion8Ji-Gang Zhang9Jessica D.S. Grassmann10Diane Luo11William F. Flynn12Elise T. Courtois13Chih-Hao Chang14David V. Serreze15Francesca Menghi16Laura G. Reinholdt17Edison T. Liu18The Jackson Laboratory for Mammalian Genetics, Bar Harbor, ME 04609, USAThe Jackson Laboratory for Mammalian Genetics, Bar Harbor, ME 04609, USAThe Jackson Laboratory, Sacramento, CA 95838, USAThe Jackson Laboratory for Genomic Medicine, Farmington, CT 06032, USAThe Jackson Laboratory for Mammalian Genetics, Bar Harbor, ME 04609, USAThe Jackson Laboratory for Genomic Medicine, Farmington, CT 06032, USAThe Jackson Laboratory, Sacramento, CA 95838, USAThe Jackson Laboratory for Mammalian Genetics, Bar Harbor, ME 04609, USAThe Jackson Laboratory for Genomic Medicine, Farmington, CT 06032, USAThe Jackson Laboratory for Mammalian Genetics, Bar Harbor, ME 04609, USASingle Cell Biology Lab, The Jackson Laboratory for Genomic Medicine, Farmington, CT 06032, USASingle Cell Biology Lab, The Jackson Laboratory for Genomic Medicine, Farmington, CT 06032, USASingle Cell Biology Lab, The Jackson Laboratory for Genomic Medicine, Farmington, CT 06032, USASingle Cell Biology Lab, The Jackson Laboratory for Genomic Medicine, Farmington, CT 06032, USA; OB/Gyn Department, UConn Health, Farmington, CT 06032, USAThe Jackson Laboratory for Mammalian Genetics, Bar Harbor, ME 04609, USAThe Jackson Laboratory for Mammalian Genetics, Bar Harbor, ME 04609, USAThe Jackson Laboratory for Genomic Medicine, Farmington, CT 06032, USAThe Jackson Laboratory for Mammalian Genetics, Bar Harbor, ME 04609, USAThe Jackson Laboratory for Genomic Medicine, Farmington, CT 06032, USA; Corresponding authorSummary: Identifying host genetic factors modulating immune checkpoint inhibitor (ICI) efficacy is experimentally challenging. Our approach, utilizing the Collaborative Cross mouse genetic resource, fixes the tumor genomic configuration while varying host genetics. We find that response to anti-PD-1 (aPD1) immunotherapy is significantly heritable in four distinct murine tumor models (H2: 0.18–0.40). For the MC38 colorectal carcinoma system, we map four significant ICI response quantitative trait loci (QTLs) with significant epistatic interactions. The differentially expressed genes within these QTLs that define responder genetics are highly enriched for processes involving antigen processing and presentation, allograft rejection, and graft vs. host disease (all p < 1 × 10−10). Functional blockade of two top candidate immune targets, GM-CSF and IL-2RB, completely abrogates the MC38 transcriptional response to aPD1 therapy. Thus, our in vivo experimental platform is a powerful approach for discovery of host genetic factors that establish the tumor immune microenvironment propitious for ICI response.http://www.sciencedirect.com/science/article/pii/S2211124725004693CP: CancerCP: Immunology
spellingShingle Daniel A. Skelly
John P. Graham
Mingshan Cheng
Mayuko Furuta
Andrew Walter
Thomas A. Stoklasek
Hongyuan Yang
Timothy M. Stearns
Olivier Poirion
Ji-Gang Zhang
Jessica D.S. Grassmann
Diane Luo
William F. Flynn
Elise T. Courtois
Chih-Hao Chang
David V. Serreze
Francesca Menghi
Laura G. Reinholdt
Edison T. Liu
Mapping the genetic landscape establishing a tumor immune microenvironment favorable for anti-PD-1 response
Cell Reports
CP: Cancer
CP: Immunology
title Mapping the genetic landscape establishing a tumor immune microenvironment favorable for anti-PD-1 response
title_full Mapping the genetic landscape establishing a tumor immune microenvironment favorable for anti-PD-1 response
title_fullStr Mapping the genetic landscape establishing a tumor immune microenvironment favorable for anti-PD-1 response
title_full_unstemmed Mapping the genetic landscape establishing a tumor immune microenvironment favorable for anti-PD-1 response
title_short Mapping the genetic landscape establishing a tumor immune microenvironment favorable for anti-PD-1 response
title_sort mapping the genetic landscape establishing a tumor immune microenvironment favorable for anti pd 1 response
topic CP: Cancer
CP: Immunology
url http://www.sciencedirect.com/science/article/pii/S2211124725004693
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