SUGAR-seq reveals the transcriptome and N-linked glycosylation landscape of mononuclear phagocytes at single-cell resolution in a mouse model of autosomal dominant osteopetrosis type 2

Abstract Background Heterozygous mutation of CLCN7 (R286W) is commonly found in patients with benign autosomal dominant osteopetrosis. However, there is no evidence from animal models to confirm that it is a disease mutation. And the characteristics of the bone marrow cell (BMC) landscape in osteope...

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Main Authors: Yu Sha, Lingyu Huang, Lei Zhang, Xianliang Hou, Chune Mo, Cuiping Pan, Gengshuo Chen, Sha Luo, Minglin Ou
Format: Article
Language:English
Published: BMC 2025-03-01
Series:BMC Biology
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Online Access:https://doi.org/10.1186/s12915-025-02193-z
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author Yu Sha
Lingyu Huang
Lei Zhang
Xianliang Hou
Chune Mo
Cuiping Pan
Gengshuo Chen
Sha Luo
Minglin Ou
author_facet Yu Sha
Lingyu Huang
Lei Zhang
Xianliang Hou
Chune Mo
Cuiping Pan
Gengshuo Chen
Sha Luo
Minglin Ou
author_sort Yu Sha
collection DOAJ
description Abstract Background Heterozygous mutation of CLCN7 (R286W) is commonly found in patients with benign autosomal dominant osteopetrosis. However, there is no evidence from animal models to confirm that it is a disease mutation. And the characteristics of the bone marrow cell (BMC) landscape in osteopetrosis at the single-cell level are completely unknown till now. Results In this study, we generated the first autosomal dominant osteopetrosis type 2 (ADO2) mouse model with typical phenotypes carried a mutation Clcn7 (r284w) corresponding to CLCN7 (R286W) observed in human patients using gene editing technology. And then, we conducted the first-ever single-cell analysis of the RNA expression and N-linked glycosylation profiles for the mouse BMCs by SUrface-protein Glycan And RNA-sequencing (SUGAR-seq). We identified 14 distinct cell types and similar proportion of neutrophils in both ADO2 and wild type mice, confirmed by flow cytometry analysis. The N-linked glycosylation modifications of BMCs were significantly downregulated detecting by SUGAR-seq, which was similar to the situation of N-Glycan profiling by the 4D Label-Free N-Glycosylation Proteomics Analysis. Particularly noteworthy is the heterogeneity of classic monocytes. We identified six cell subtypes, but only two cell subtypes were found with different proportion of cell, whose different expressed genes were associated with NF-κB-inducing kinase / Nuclear Factor-kappa B (NIK/NF-κB) signaling and other pathway associated with osteoclast differentiation. Conclusions Our murine model confirms that the human CLCN7 (R286W) is a pathogenic mutation for ADO2. Additionally, our single-cell analyses reveal the heterogeneity of monocytes in ADO2, and the abnormal glycosylation modifications across various subtypes may represent important events in the pathogenesis of osteopetrosis.
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spelling doaj-art-a7e5c3465e9345f1a5ab67d44f4bc5ad2025-08-20T03:07:44ZengBMCBMC Biology1741-70072025-03-0123111910.1186/s12915-025-02193-zSUGAR-seq reveals the transcriptome and N-linked glycosylation landscape of mononuclear phagocytes at single-cell resolution in a mouse model of autosomal dominant osteopetrosis type 2Yu Sha0Lingyu Huang1Lei Zhang2Xianliang Hou3Chune Mo4Cuiping Pan5Gengshuo Chen6Sha Luo7Minglin Ou8Laboratory Center, Guangxi Key Laboratory of Metabolic Reprogramming and Intelligent Medical Engineering for Chronic Diseases, The Second Affiliated Hospital of Guilin Medical UniversityLaboratory Center, Guangxi Key Laboratory of Metabolic Reprogramming and Intelligent Medical Engineering for Chronic Diseases, The Second Affiliated Hospital of Guilin Medical UniversityThe Department of Nuclear Medicine, Affiliated Hospital of Guilin Medical UniversityLaboratory Center, Guangxi Key Laboratory of Metabolic Reprogramming and Intelligent Medical Engineering for Chronic Diseases, The Second Affiliated Hospital of Guilin Medical UniversityLaboratory Center, Guangxi Key Laboratory of Metabolic Reprogramming and Intelligent Medical Engineering for Chronic Diseases, The Second Affiliated Hospital of Guilin Medical UniversityLaboratory Center, Guangxi Key Laboratory of Metabolic Reprogramming and Intelligent Medical Engineering for Chronic Diseases, The Second Affiliated Hospital of Guilin Medical UniversityLaboratory Center, Guangxi Key Laboratory of Metabolic Reprogramming and Intelligent Medical Engineering for Chronic Diseases, The Second Affiliated Hospital of Guilin Medical UniversityLaboratory Center, Guangxi Key Laboratory of Metabolic Reprogramming and Intelligent Medical Engineering for Chronic Diseases, The Second Affiliated Hospital of Guilin Medical UniversityLaboratory Center, Guangxi Key Laboratory of Metabolic Reprogramming and Intelligent Medical Engineering for Chronic Diseases, The Second Affiliated Hospital of Guilin Medical UniversityAbstract Background Heterozygous mutation of CLCN7 (R286W) is commonly found in patients with benign autosomal dominant osteopetrosis. However, there is no evidence from animal models to confirm that it is a disease mutation. And the characteristics of the bone marrow cell (BMC) landscape in osteopetrosis at the single-cell level are completely unknown till now. Results In this study, we generated the first autosomal dominant osteopetrosis type 2 (ADO2) mouse model with typical phenotypes carried a mutation Clcn7 (r284w) corresponding to CLCN7 (R286W) observed in human patients using gene editing technology. And then, we conducted the first-ever single-cell analysis of the RNA expression and N-linked glycosylation profiles for the mouse BMCs by SUrface-protein Glycan And RNA-sequencing (SUGAR-seq). We identified 14 distinct cell types and similar proportion of neutrophils in both ADO2 and wild type mice, confirmed by flow cytometry analysis. The N-linked glycosylation modifications of BMCs were significantly downregulated detecting by SUGAR-seq, which was similar to the situation of N-Glycan profiling by the 4D Label-Free N-Glycosylation Proteomics Analysis. Particularly noteworthy is the heterogeneity of classic monocytes. We identified six cell subtypes, but only two cell subtypes were found with different proportion of cell, whose different expressed genes were associated with NF-κB-inducing kinase / Nuclear Factor-kappa B (NIK/NF-κB) signaling and other pathway associated with osteoclast differentiation. Conclusions Our murine model confirms that the human CLCN7 (R286W) is a pathogenic mutation for ADO2. Additionally, our single-cell analyses reveal the heterogeneity of monocytes in ADO2, and the abnormal glycosylation modifications across various subtypes may represent important events in the pathogenesis of osteopetrosis.https://doi.org/10.1186/s12915-025-02193-zOsteopetrosisSingle-cell resolutionMononuclear phagocytesGlycoproteome
spellingShingle Yu Sha
Lingyu Huang
Lei Zhang
Xianliang Hou
Chune Mo
Cuiping Pan
Gengshuo Chen
Sha Luo
Minglin Ou
SUGAR-seq reveals the transcriptome and N-linked glycosylation landscape of mononuclear phagocytes at single-cell resolution in a mouse model of autosomal dominant osteopetrosis type 2
BMC Biology
Osteopetrosis
Single-cell resolution
Mononuclear phagocytes
Glycoproteome
title SUGAR-seq reveals the transcriptome and N-linked glycosylation landscape of mononuclear phagocytes at single-cell resolution in a mouse model of autosomal dominant osteopetrosis type 2
title_full SUGAR-seq reveals the transcriptome and N-linked glycosylation landscape of mononuclear phagocytes at single-cell resolution in a mouse model of autosomal dominant osteopetrosis type 2
title_fullStr SUGAR-seq reveals the transcriptome and N-linked glycosylation landscape of mononuclear phagocytes at single-cell resolution in a mouse model of autosomal dominant osteopetrosis type 2
title_full_unstemmed SUGAR-seq reveals the transcriptome and N-linked glycosylation landscape of mononuclear phagocytes at single-cell resolution in a mouse model of autosomal dominant osteopetrosis type 2
title_short SUGAR-seq reveals the transcriptome and N-linked glycosylation landscape of mononuclear phagocytes at single-cell resolution in a mouse model of autosomal dominant osteopetrosis type 2
title_sort sugar seq reveals the transcriptome and n linked glycosylation landscape of mononuclear phagocytes at single cell resolution in a mouse model of autosomal dominant osteopetrosis type 2
topic Osteopetrosis
Single-cell resolution
Mononuclear phagocytes
Glycoproteome
url https://doi.org/10.1186/s12915-025-02193-z
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