Cited by 230Down syndrome and intellectual disability researchinterferon and immune responsesImmunodeficiency and Autoimmune Disorders

The DOI matches its Crossref record; 95 of the 95 references it cites were found in a registry or an index; 13 of the 41 sources its claims cite were checked for support.

Trisomy 21 causes changes in the circulating proteome indicative of chronic autoinflammation

Open access

Abstract

Trisomy 21 (T21) causes Down syndrome (DS), but the mechanisms by which T21 produces the different disease spectrum observed in people with DS are unknown. We recently identified an activated interferon response associated with T21 in human cells of different origins, consistent with overexpression of the four interferon receptors encoded on chromosome 21, and proposed that DS could be understood partially as an interferonopathy. However, the impact of T21 on systemic signaling cascades in living individuals with DS is undefined. To address this knowledge gap, we employed proteomics approaches to analyze blood samples from 263 individuals, 165 of them with DS, leading to the identification of dozens of proteins that are consistently deregulated by T21. Most prominent among these proteins are numerous factors involved in immune control, the complement cascade, and growth factor signaling. Importantly, people with DS display higher levels of many pro-inflammatory cytokines (e.g. IL-6, MCP-1, IL-22, TNF-α) and pronounced complement consumption, resembling changes seen in type I interferonopathies and other autoinflammatory conditions. Therefore, these results are consistent with the hypothesis that increased interferon signaling caused by T21 leads to chronic immune dysregulation, and justify investigations to define the therapeutic value of immune-modulatory strategies in DS.

Overview

AI snapshot

Written from the paper’s full text.

TL;DR

Trisomy 21 is associated with widespread changes in the circulating proteome, including immune-control, complement/coagulation, growth-factor, and neurogenesis-related proteins. The pattern includes elevated pro-inflammatory cytokines and complement consumption, resembling type I interferonopathies and supporting chronic autoinflammation in Down syndrome.

Key findings

  • In the Discovery Study (120 T21, 52 D21; 3,585 epitopes), 299 proteins were differentially detected at Bonferroni-adjusted p < 0.1: 178 downregulated and 121 upregulated in T21. Among 50 chr21-encoded proteins with aptamers, 9 were significantly upregulated and none significantly downregulated.
  • Validation was reproducible: ~72% of Discovery differential proteins measured in all three studies validated in at least one Validation Study (FDR < 10%). B2M was consistently elevated and IGHE consistently downregulated; lower IgE was confirmed by ELISA.
  • In a separate MSD assay of 40 adults (20 T21, 20 D21), individuals with DS had significantly increased IL-6, IL-22, MCP-1/CCL2, TNF-α, and VEGF-A. CCL17 was elevated in all three SOMAscan studies but fell below the MSD significance cutoff.
  • Complement/coagulation proteins showed a consumption pattern: 21 proteins in the category, 18 downregulated in DS, including C1R, C1QA, C3, and C6; C1S was consistently depleted in all three studies. Interferome mining found significant enrichment of interferon-related proteins among differential proteins (p = 1.91E-03, hypergeometric test).

Methods

  • SOMAscan aptamer proteomics was performed on plasma/serum: Discovery Study (172 individuals: 120 T21, 52 D21; 3,585 epitopes), Validation Study #1 (53: 38 T21, 15 D21; plasma; 1,047 epitopes), and Validation Study #2 (33: 22 T21, 11 D21; serum; 1,047 epitopes). The abstract reports 263 individuals total, 165 with DS.
  • A Mesoscale Discovery assay measured 38 cytokines in plasma from 40 adults (20 T21, 20 D21; ages 20–65), and IgE ELISA was used for confirmation.
  • Differential detection used the non-parametric Kolmogorov-Smirnov test with Bonferroni correction for the Discovery Study and Benjamini-Hochberg correction for smaller Validation/MSD studies; functional analyses used Metascape, STRING, and Interferome.

Limitations

  • The paper does not state a formal limitations section; it describes the proteomics datasets as “hypothesis-generating” and calls for future mechanistic investigations.
  • Evident from the text: the SOMAscan cohorts are largely pediatric (Discovery ages six months to 21 years), while the MSD cohort is only 40 adults (20–65), so age coverage is limited and the design is cross-sectional.
  • Evident from the text: no direct measurement of interferon signaling or functional validation is reported, and the findings are associations in the circulating proteome; causality and therapeutic benefit of immune modulation are not tested.

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Integrity signals

Registry

Crossref · 10.1038/s41598-017-13858-3

Cites

95 of 95 references verified0 citation errors · 0 not checkable

Statistics

Nothing to recompute.

Figures

Figure 1 Individuals with trisomy 21 display consistent differences in their circulating proteomes. (a) Volcano plots displaying the results of a SOMAscan® proteomics analysis for 3,585 epitopes detected in plasma samples from individuals with or without trisomy 21 (T21). This cohort, referred to as the Discovery Study, involved 120 individuals with T21 and 52 euploid (D21) controls. Adjusted p-values [p(a)] were generated with the Kolmogorov-Smirnov test using a Bonferroni correction for multiple hypothesis testing. When using a cut-off of p(a) <0.1, 178 proteins were identified as significantly downregulated in people with T21 (e.g. Immunoglobulin E, IGHE) versus 121 upregulated proteins (e.g. β-2-microglobulin, B2M). Green dots indicate the 50 proteins encoded in chromosome 21 (chr21) for which aptamers were available in the SOMAscan® assay, only 9 of which passed the p(a) <0.1 cut-off (e.g. TFF3). (b) For comparison purposes, data in (A) was re-analyzed to identify differences between females and males. KLK3, downregulated in females, is the prostate-specific antigen (PSA). CGA, upregulated in females, is the alpha subunit of the follicle stimulating and luteinizing hormones. (c) Manhattan plot of all detected proteins showing that most differential proteins observed are not encoded on chr21 (top). Individual Manhattan plots showing the proteins encoded on chr14, chr15 and chr21 are shown at the bottom. Red dashed line indicates a zero-fold change. Significantly different proteins are defined as p(a) < 0.1 using KS test with Bonferroni correction. (d) Box and whisker plots showing the comparative results for B2M and IGHE in the Discovery Study and two smaller Validation Studies. Adjusted p-values shown from KS test with Bonferroni correction for the Discovery Study and Benjamini-Hochberg for the smaller Validation Studies. See also Figs 1S1 and S2. (e) Metascape analysis of significantly differential proteins in the Discovery Study, as defined by p(a) <0.1, 299 proteins in total. Each node represents a GO term, KEGG pathway, or Reactome gene set. The 3,585 proteins detected by SOMAscan® assay were used as the background gene set. See also Figs 1S3, 1S4, and Supplementary File 2.
Figure 1 Individuals with trisomy 21 display consistent differences in their circulating proteomes. (a) Volcano plots displaying the results of a SOMAscan® proteomics analysis for 3,585 epitopes detected in plasma samples from individuals with or without trisomy 21 (T21). This cohort, referred to as the Discovery Study, involved 120 individuals with T21 and 52 euploid (D21) controls. Adjusted p-values [p(a)] were generated with the Kolmogorov-Smirnov test using a Bonferroni correction for multiple hypothesis testing. When using a cut-off of p(a) <0.1, 178 proteins were identified as significantly downregulated in people with T21 (e.g. Immunoglobulin E, IGHE) versus 121 upregulated proteins (e.g. β-2-microglobulin, B2M). Green dots indicate the 50 proteins encoded in chromosome 21 (chr21) for which aptamers were available in the SOMAscan® assay, only 9 of which passed the p(a) <0.1 cut-off (e.g. TFF3). (b) For comparison purposes, data in (A) was re-analyzed to identify differences between females and males. KLK3, downregulated in females, is the prostate-specific antigen (PSA). CGA, upregulated in females, is the alpha subunit of the follicle stimulating and luteinizing hormones. (c) Manhattan plot of all detected proteins showing that most differential proteins observed are not encoded on chr21 (top). Individual Manhattan plots showing the proteins encoded on chr14, chr15 and chr21 are shown at the bottom. Red dashed line indicates a zero-fold change. Significantly different proteins are defined as p(a) < 0.1 using KS test with Bonferroni correction. (d) Box and whisker plots showing the comparative results for B2M and IGHE in the Discovery Study and two smaller Validation Studies. Adjusted p-values shown from KS test with Bonferroni correction for the Discovery Study and Benjamini-Hochberg for the smaller Validation Studies. See also Figs 1S1 and S2. (e) Metascape analysis of significantly differential proteins in the Discovery Study, as defined by p(a) <0.1, 299 proteins in total. Each node represents a GO term, KEGG pathway, or Reactome gene set. The 3,585 proteins detected by SOMAscan® assay were used as the background gene set. See also Figs 1S3, 1S4, and Supplementary File 2.CC BY · PubMed Central
Figure 2 People with Down syndrome show a proteomic signature indicative of profound immune dysregulation. (a) Metascape analysis highlighting interconnected protein networks involved in Immune Control among the differential proteins in the Discovery Study. (b) STRING analysis identifies interacting groups of deregulated proteins within the Immune Control functional category. (c–j) Box and whisker plots of SOMAscan® data for individual proteins deregulated in individuals with T21. p(a) values from KS test with Bonferroni correction. (k) Heatmap displaying the results of a Mesoscale Discovery Assay (MSD) measuring the levels of 38 inflammatory markers in a cohort of 40 adult individuals, 20 of them with T21. Proteins are ranked from the most significantly different (top, IL-6) to not significantly different (bottom, IL-1α). The horizontal dashed line marks the p(a) <0.1 cut-off. (l–p) Box and whisker plots for the top five most different inflammatory markers detected in the MSD assay, all of which are elevated in people with DS. p(a) values from KS test with Benjamini-Hochberg correction.
Figure 2 People with Down syndrome show a proteomic signature indicative of profound immune dysregulation. (a) Metascape analysis highlighting interconnected protein networks involved in Immune Control among the differential proteins in the Discovery Study. (b) STRING analysis identifies interacting groups of deregulated proteins within the Immune Control functional category. (c–j) Box and whisker plots of SOMAscan® data for individual proteins deregulated in individuals with T21. p(a) values from KS test with Bonferroni correction. (k) Heatmap displaying the results of a Mesoscale Discovery Assay (MSD) measuring the levels of 38 inflammatory markers in a cohort of 40 adult individuals, 20 of them with T21. Proteins are ranked from the most significantly different (top, IL-6) to not significantly different (bottom, IL-1α). The horizontal dashed line marks the p(a) <0.1 cut-off. (l–p) Box and whisker plots for the top five most different inflammatory markers detected in the MSD assay, all of which are elevated in people with DS. p(a) values from KS test with Benjamini-Hochberg correction.CC BY · PubMed Central
Figure 3 People with Down syndrome exhibit hypocomplementia. (a) Metascape analysis highlighting interconnected protein networks related to Complement and Coagulation among the differential proteins in the Discovery Study. (b) STRING analysis identifying interacting groups of deregulated proteins within the Complement and Coagulation functional category. (c–j) Box and whisker plot showing data for individual proteins. p(a) values from KS test with Bonferroni correction.
Figure 3 People with Down syndrome exhibit hypocomplementia. (a) Metascape analysis highlighting interconnected protein networks related to Complement and Coagulation among the differential proteins in the Discovery Study. (b) STRING analysis identifying interacting groups of deregulated proteins within the Complement and Coagulation functional category. (c–j) Box and whisker plot showing data for individual proteins. p(a) values from KS test with Bonferroni correction.CC BY · PubMed Central
Figure 4 People with Down syndrome show different levels of proteins involved in growth factor signaling and control of neurogenesis. (a) Metascape analysis highlighting protein networks related to Growth Factor Signaling and Regulation of Neurogenesis among the differential proteins in the Discovery Study. (b) STRING analysis identifying interacting groups of deregulated proteins within the Growth Factor Signaling category. (c–n) Box and whisker plots displaying SOMAscan® data for individual proteins deregulated in individuals with T21. p(a) from KS test with Bonferroni correction.
Figure 4 People with Down syndrome show different levels of proteins involved in growth factor signaling and control of neurogenesis. (a) Metascape analysis highlighting protein networks related to Growth Factor Signaling and Regulation of Neurogenesis among the differential proteins in the Discovery Study. (b) STRING analysis identifying interacting groups of deregulated proteins within the Growth Factor Signaling category. (c–n) Box and whisker plots displaying SOMAscan® data for individual proteins deregulated in individuals with T21. p(a) from KS test with Bonferroni correction.CC BY · PubMed Central

Verification

Claims the paper makes

44 Own result · 13 Own interpretation · 12 No full text of the sources · 8 Own method · 6 Partly supported · 4 Supported · 1 Supported so far · 1 Not checked yet · accuracy not yet measured

Show all 51 claims

  • Probably not a substantial claim. 25
  • It may state two separate claims as one. 18
38 of 38
  • The paper relies on this without support.

    Trisomy 21 causes Down syndrome.

    “Trisomy 21 (T21) causes Down syndrome (DS)”
  • The paper relies on this without support.

    The mechanisms by which trisomy 21 produces the different disease spectrum observed in people with Down syndrome are unknown.

    “the mechanisms by which T21 produces the different disease spectrum observed in people with DS are unknown”
  • The paper relies on this without support.

    The impact of trisomy 21 on systemic signaling cascades in living individuals with Down syndrome is undefined.

    “the impact of T21 on systemic signaling cascades in living individuals with DS is undefined”
  • The paper relies on this without support.

    The four interferon receptors encoded on chromosome 21 are overexpressed in trisomy 21.

    “consistent with overexpression of the four interferon receptors encoded on chromosome 21”
4 of 4

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Metadata

Authors
Sullivan, K. D., Evans, D., Pandey, A., Hraha, T. H., Smith, K. P., Markham, N., Rachubinski, A. L., Wolter-Warmerdam, K., Hickey, F., Espinosa, J. M., & Blumenthal, T.
Affiliations
  • University of Colorado Anschutz (US)
  • SomaLogic (United States) (US)
  • Children's Hospital Colorado (US)
  • University of Colorado Boulder (US)
Corresponding author
  • Espinosa, Joaquin M.
  • Blumenthal, Thomas
Venue
Scientific Reports
Published
2017-11-01
Volume
7
Issue
1
Pages
14818-14818
ISSN
2045-2322 (ISSN-L)
OpenAlex
W2766826848
Cited by
228
References
95
Citations by year
2026: 23 · 2025: 21 · 2024: 33 · 2023: 29 · 2022: 34 · 2021: 29 · 2020: 31 · 2019: 19 · 2018: 9
FWCI
13.90
Type
journal article
Language
en
Open access
Locations
  • Scientific Reports (publishedVersion, cc-by)
  • PubMed (publishedVersion)
  • PubMed Central (submittedVersion, cc-by)
  • DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion)
Funders
  • Global Down Syndrome Foundation
  • Anna and John J. Sie Foundation
  • Linda Crnic Institute for Down Syndrome, University of Colorado School of Medicine, Anschutz Medical Campus
Topics
  • Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Down syndrome and intellectual disability research
  • Life Sciences › Immunology and Microbiology › Immunology › interferon and immune responses
  • Life Sciences › Immunology and Microbiology › Immunology › Immunodeficiency and Autoimmune Disorders
Keywords
Down syndrome, circulating proteome, interferonopathies, type I interferon signaling, interferon receptor, complement system, inflammatory cytokines, immune dysregulation, growth factor signaling, immunomodulatory therapy
MeSH terms
Adolescent, Adult, Child, Child, Preschool, Chronic Disease, Complement System Proteins, Down Syndrome, Female, Humans, Infant, Inflammation, Male, Trisomy, Cytokines, Receptors, Growth Factor, Proteome, Young Adult

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