bims-hummad Biomed News
on Humanised mouse models of autoimmune disorders
Issue of 2026–06–14
nine papers selected by
Maksym V. Kopanitsa, Charles River Laboratories



  1. Front Immunol. 2026 ;17 1793493
       Background: Sjögren's syndrome (SS) is a systemic autoimmune disease characterized by lymphocytic infiltration of exocrine glands, leading to impaired glandular secretion. To elucidate the pathogenic mechanisms underlying SS, suitable preclinical animal models are essential. In this study, we developed a humanized murine model that captures key immunopathological features of SS patients, and assessed its therapeutic utility.
    Methods: PBMCs obtained from SS patients were stimulated with anti-CD3 and anti-CD28 antibodies for 15 hours, and 1 × 106 or 2 × 106 of these cells were intraperitoneally injected into NOD.Cg-Prkdc scid Il2rg tm1Wjl/SzJ (NSG) mice. At 5 weeks after cell injection, pathological analysis and immunophenotypic characterization of infiltrating immune cells within the salivary gland tissues were performed. To evaluate the efficacy of metformin, NSG mice transplanted with PBMCs were orally administered metformin daily for 5 weeks.
    Results: Mice injected with PBMCs from SS patients exhibited a significant increase in the frequency of human IL-17-producing T cells in the spleen, accompanied by enhanced infiltration of these pro-inflammatory cells into the salivary glands. Histopathological analysis of salivary glands revealed marked immune cell infiltration and a significant reduction in Aquaporin-5 expression in SS-derived PBMC-injected mice. Notably, these pathological changes were associated with the local recruitment of CXCR3+ Th17 cells. Metformin treatment significantly attenuated salivary gland inflammation, reduced the infiltration of pathogenic T cells, and mitigated molecular-level tissue damage in this humanized SS model.
    Conclusions: The humanized murine model developed in this study effectively reproduced key cellular and molecular features of SS and provided a useful preclinical platform for investigating early-stage disease mechanisms and evaluating novel therapeutic strategies for SS.
    Keywords:  CXCR3; IL-17-producing T cells; NSG mouse; Sjogren’s syndrome; humanized mouse
    DOI:  https://doi.org/10.3389/fimmu.2026.1793493
  2. J Invest Dermatol. 2026 Jun 12. pii: S0022-202X(26)02602-3. [Epub ahead of print]
      Alopecia areata (AA) is a chronic autoimmune disease characterized by sudden patchy hair loss and persistent inflammation. To date, only JAK inhibitors have been approved for AA treatment, but FDA-issued black box warnings highlight the need for alternative therapies. We previously reported that an anti-γc antibody, hC2, inhibits autoreactive B, T, and NK cells by selectively attenuating JAK/STAT signaling induced by six γc cytokines, without affecting off-target TEC kinase pathways. Here, we sought to define the mechanism of action and efficacy of hC2 in AA by using an ex-vivo T cell platform and a xenogeneic AA-like mouse model induced by human T cell engraftment. Analyses showed that hC2 could restore hair follicle homeostasis and suppress hair loss by inhibiting autoreactive T cell activity and proliferation of tissue-resident memory T cells. Although JAK3 inhibitor ritlecitinib could potentially protect hair follicles through T cell depletion strategy in-vitro, severe side effects were associated with ritlecitinib treatment while no significant safety issues were noted after hC2 treatment in the xenogeneic AA-like mouse model. These findings suggest that hC2 might offer a safer and more effective therapeutic approach for AA patients in the future.
    Keywords:  Alopecia; Biologics; Cytokines; Immunology; T Cells
    DOI:  https://doi.org/10.1016/j.jid.2026.05.028
  3. bioRxiv. 2026 Jun 07. pii: 2026.06.03.729692. [Epub ahead of print]
      Immune-related adverse events (irAEs), particularly colitis, are major limitations of immune checkpoint inhibitor (ICI) therapy, but their mechanisms remain poorly understood. Here we show that endogenous autoantibodies (AAbs) can promote ICI-associated colitis through Fcγ receptor-dependent pathways. IgG from melanoma patients treated with pembrolizumab, nivolumab, or ipilimumab, with or without severe colitis, was transferred into wild-type or humanized FcγR (hFcγR) mice receiving comparable ICI therapy. Wild-type mice did not develop changes in the colon. In contrast, hFcγR mice given IgG from patients with colitis developed colon inflammation marked by a significant increase in submucosal lymphocyte infiltration, goblet cell loss, and circulating cytokines, including IL-1β, IL-17a, and IL-22. Single-cell RNA sequencing identified an IgG-regulated inflammatory network involving IFNγ-producing ILC1, Th1 and cytotoxic T cells, IL-1β+ M1 macrophages, plasma B cells/plasmablasts, and IL-22-producing ILC3-LTi cells. Patient serum autoantibody profiling further identified CCR5 and CXCR4 receptors as candidate immune-related targets associated with ICC susceptibility. Immune-related adverse events (irAEs), particularly colitis, are major limitations of immune checkpoint inhibitor (ICI) therapy, but their mechanisms remain poorly understood. Here we show that endogenous autoantibodies (AAbs) can promote ICI-associated colitis through Fc gamma receptor (FcgR)-dependent pathways. IgG from melanoma patients treated with pembrolizumab, nivolumab, or ipilimumab, with or without severe colitis, was transferred into wild-type or humanized FcgR (hFcgR) mice receiving comparable ICI therapy. Wild-type mice did not develop changes in the colon. In contrast, hFcgR mice given IgG from patients with colitis developed colon inflammation marked by a significant increase in submucosal lymphocyte infiltration, goblet cell loss, and circulating cytokines, including IL-6, IL-17, and IL-22. Single-cell RNA sequencing identified an IgG-regulated inflammatory network involving IFNg-producing ILC1, Th1 and cytotoxic T cells, IL-1betta-M1 macrophages, plasma B cells/plasmablasts, and IL-22-producing ILC3-LTi cells. Patient serum autoantibody profiling further identified CCR5 and CXCR4 receptors as candidate immune-related targets associated with ICC susceptibility.
    DOI:  https://doi.org/10.64898/2026.06.03.729692
  4. JCI Insight. 2026 Jun 08. pii: e200738. [Epub ahead of print]11(11):
      Stem cell-derived β cells offer a promising approach for type 1 diabetes (T1D) treatment. However, the processes of graft infiltration and rejection by immune cells remain poorly understood in humans. In this study, autologous or allogeneic stem cell-derived islets (SC-islets) were transplanted in human immune system mice and analyzed 14 to 18 weeks later. Imaging mass cytometry revealed unique characteristics of SC-islet grafts, including a high percentage of glucagon+ cells and the presence of cysts and CD57+ enterochromaffin cells, features not typically observed in endogenous or transplanted allogeneic primary pancreatic islets. Allogeneic SC-islet grafts exhibited heavy immune infiltration, cell proliferation, and pro-fibrotic processes, whereas autologous grafts showed minimal infiltration and little fibrosis. In some mice, autologous T cells expressing islet antigen-reactive (IAR) T cell receptors (TCRs) were adoptively transferred. Three weeks after transfer, autologous grafts injected with IAR-TCR+ T cells showed negligible immune infiltration, even though IAR-TCR+ T cells were detected in the spleen. Under the conditions tested, human SC-islet grafts were not rejected by an autologous immune system, even in the presence of autoreactive T cells, pointing to several limitations that remain to be addressed for a model of spontaneous autologous SC-islet infiltration and destruction.
    Keywords:  Autoimmunity; Beta cells; Endocrinology; Human stem cells; Immunology; T cells
    DOI:  https://doi.org/10.1172/jci.insight.200738
  5. J Immunother Cancer. 2026 Jun 12. pii: e014890. [Epub ahead of print]14(6):
       BACKGROUND: Relapse and graft-versus-host disease (GvHD) remain primary causes of treatment failure in patients with B-cell acute lymphoblastic leukemia (B-ALL) undergoing allogeneic hematopoietic cell transplantation (allo-HCT). While abatacept (ABATA) effectively mitigates GvHD via CD28-costimulation blockade, there is significant concern that it concurrently diminishes graft-versus-leukemia (GvL) effects, potentially leading to higher relapse rates. We investigated whether blinatumomab (BLINA) retains antileukemic efficacy in the presence of ABATA using in vitro assays and humanized NOD.Cg-Prkdcscid Il2rgtm1Wjl Tg(IL15)1Sz/SzJ (NSG-IL15) and NOD.Cg-Prkdcscid Il2rgtm1Wjl/SzJ (NSG) mouse models.
    METHODS: In vitro and ex vivo flow cytometry-based cytotoxicity, degranulation (CD107a), cytokine production (interferon (IFN)-γ), and activation-marker (CD25, CD69, CD137, OX40) assays were performed using healthy donor peripheral blood mononuclear cells (PBMCs) (pretreated±ABATA) against leukemia or lymphoma cell lines (RS4;11, NALM-6, and JEKO-1) in the presence or not of BLINA. In vivo human PBMC-reconstituted NSG-IL-15 mice bearing RS4;11 B-ALL xenografts were treated with vehicle, monotherapy (BLINA or ABATA), or combination therapy. Leukemia burden and immune reconstitution were assessed through week 4; GvHD scores and overall survival were monitored longitudinally.
    RESULTS: ABATA did not impair BLINA-mediated cytotoxicity across multiple effector-to-target ratios against any of the cell lines, nor did it reduce BLINA-induced degranulation, IFN-γ production, or activation marker expression. In vivo, BLINA monotherapy significantly reduced leukemia burden, but resulted in early mortality due to severe GvHD. Conversely, ABATA markedly reduced GvHD severity. The combination of BLINA and ABATA preserved potent antileukemic efficacy while significantly extending median survival by 35 days compared with tumor controls (p<0.0001). Although ABATA attenuated T-cell expansion and differentiation, BLINA-driven cytotoxic function was maintained, even after prolonged in vivo exposure to cytotoxic T-lymphocyte-associated protein 4-immunoglobulin (CTLA-4-Ig).
    CONCLUSION: These findings provide preclinical proof-of-concept that BLINA and ABATA can be combined to uncouple GvL from GvHD. This strategy preserves CD28-independent T-cell cytotoxicity while limiting allo-reactivity, providing a strong rationale for investigating this combination in the post-transplant setting.
    Keywords:  Bispecific T cell engager - BiTE; Graft versus host disease - GVHD; Graft versus leukemia; Immunotherapy; Leukemia
    DOI:  https://doi.org/10.1136/jitc-2026-014890
  6. Transplantation. 2026 Jun 09.
       BACKGROUND: Despite being promising candidates for treating acute graft-versus-host disease (aGvHD), the efficacy and underlying mechanisms of mesenchymal stem cells (MSCs) may vary based on their sources.
    METHODS: T-MSCs, derived from trophoblast cells, and BM-MSCs, derived from bone marrow cells, were investigated for their efficacy in alleviating aGvHD in humanized NOD.Cg-Prkdcscid Il2rgtm1vst/Vst (NPG) mice. Clinical scoring and pathological damage were evaluated. Flow cytometry and cytometric bead array were used to assess T-cell proliferation and related cytokine release. Mechanisms involved in MSC-related efficacy were probed using different inhibitors and blocking antibodies.
    RESULTS: An acute, single dose of T-MSCs (3 × 105 cells/mouse) or BM-MSCs (3 × 105 cells/mouse) significantly ameliorated aGvHD severity, whereas delayed, multiple doses of either MSC type did not. At the cellular level, both T-MSCs and BM-MSCs inhibited the proliferation of CD4+ and CD8+ T cells and the upregulation of related cytokines, possibly involving prostaglandin E2 but not indoleamine 2,3-dioxygenase 1, transforming growth factor-β, or the programmed death-ligand 1 or Fas signaling. Additionally, the CD3+CD25high+Foxp3+ T cells were increased by both MSC types.
    CONCLUSIONS: Both T-MSCs and BM-MSCs can prevent CD4+ and CD8+ T-cell proliferation, possibly by facilitating prostaglandin E2 release and promoting the proliferation of CD3+CD25high+Foxp3+ T cells, thereby alleviating the development of aGvHD. T-MSCs and BM-MSCs may serve as promising candidates for treating aGvHD in the clinical setting.
    DOI:  https://doi.org/10.1097/TP.0000000000005781
  7. Arthritis Rheumatol. 2026 Jun 08.
       OBJECTIVE: Anti-homocitrullinated protein/peptide antibodies are specific to rheumatoid arthritis (RA) and predictive of worse prognosis, suggesting a pathogenic role for autoreactivity to homocitrullinated peptides (HomoCitP). However, T-cell responses to HomoCitP remain largely unexplored. We investigated these responses in a humanized HLA-DR4-transgenic (DR4tg) mouse model of RA, which expresses the strongest genetic risk factor for this disease.
    METHODS: DR4tg mice (n=6-15) were injected subcutaneously with a HomoCitP called HomoCitJED while control mice received phosphate-buffered saline. After 10 days, T-cells were analyzed for their phenotypic characteristics, cytokine production, and proliferative capacities in draining lymph nodes (dLNs) and spleens by flow cytometry, enzyme-linked immunosorbent assays, and ProQuantum™ immunoassays.
    RESULTS: HomoCitJED immunization, compared with controls, drove robust expansion of T helper subsets in the dLNs, including more than a 7-fold increase in Th1, Th17, and hybrid Th1/Th17 cells (p<0.0087). Cytokine staining confirmed effector activity, with higher frequencies of IL-17A+, TNF-α+IL-17A+, and IFN-γ+IL-17A+ cells (p<0.0207). Exhaustion-associated markers were also increased, including LAG-3+ and Tim-3+ CD4+ T cells (p<0.0411). CD8+ T cells mirrored these findings, with a 6-fold increase in CD25 and doubled KLRG1 expression, accompanied by augmented IL-17A and IFN-γ/IL-17A production and antigen-specific proliferation (p<0.0379). Expression of exhaustion-associated markers Tim-3+ and LAG-3+ on CD8+ T cells was likewise elevated (p<0.0205).
    CONCLUSIONS: HomoCitP elicited Th1/Th17 and Tc1/Tc17 responses marked by concurrent expression of activation and exhaustion-associated markers, consistent with a dysregulated state. These findings position homocitrulline-driven CD4+ and CD8+ T-cell imbalance as a potential mechanistic contributor to RA pathogenesis and a target for future immunomodulatory therapies.
    DOI:  https://doi.org/10.1002/art.70239
  8. JHEP Rep. 2026 Jun 12. pii: S2589-5559(26)00196-5. [Epub ahead of print] 101925
       BACKGROUND & AIMS: Primary sclerosing cholangitis (PSC) is an immune-mediated liver disease with an incompletely understood pathogenesis. Monocyte/macrophage cell dysregulation has been suggested to contribute, although their functional relevance remains insufficiently understood. The main aim of this study was to identify peripheral myeloid cell populations involved in PSC pathogenesis and assess the therapeutic potential of targeting associated inflammatory signalling pathways. Additionally, we sought to enhance translational relevance through the application of a humanized immune system (HIS) mouse model.
    METHODS: Peripheral blood mononuclear cells from PSC and ulcerative colitis patients and from healthy controls were profiled using cellular indexing of transcriptomes and epitopes by sequencing. Inflammatory signalling via the IL-8:CXCR1/2 axis was further evaluated in both conventional and HIS mouse models of cholestatic liver disease.
    RESULTS: Our data show that PSC patients exhibit a population of circulatory CXCL8+CD14+ monocytes with upregulated proinflammatory signalling. Serum concentrations of the proinflammatory cytokine IL-8, encoded by CXCL8, were increased in PSC patients (p<0.001) and associated with poor prognosis. PSC patients with high IL-8 concentrations (≥ 27.8 pg/mL) at baseline had a significantly worse transplant-free survival in comparison to patients with low IL-8 concentrations (logrank hazard ratio 6.12, 95% CI 1.68-22.33). Intrahepatic IL-8+ macrophages were expanded during disease progression (p<0.05). Pharmacologic Cxcr1 antagonism in conventional rodent immune system and antibody-mediated IL-8 neutralization in HIS mice reduced cholestatic liver injury.
    CONCLUSIONS: A circulatory CXCL8+CD14+ monocyte subset and intrahepatic IL-8+ macrophages were identified as potential contributors to PSC pathogenesis. HIS mouse models could mark a significant advance in translational research on human immune responses. Targeting the IL-8:CXCR1/2 axis may represent a promising therapeutic strategy and urges further investigation.
    Keywords:  Cholestatic liver disease; Humanized Immune System Mouse Model; Macrophage; Pro-inflammatory cytokine; Single-Cell Analysis
    DOI:  https://doi.org/10.1016/j.jhepr.2026.101925
  9. JCI Insight. 2026 Jun 08. pii: e200928. [Epub ahead of print]11(11):
      Synovial hyperplasia is a hallmark of rheumatoid arthritis (RA), yet its mechanism remains unclear. RA synovium exhibits metabolic shift, characterized by upregulated glycolysis and enhanced lactate production. In this study, we elucidated the mechanism underlying the roles of lactate metabolism and protein lactylation in RA pathology. In patients with RA, both lactate production and protein lactylation were elevated and showed a positive correlation with clinical disease activity. These changes were further implicated in driving synovial proliferation. Among the lactylated proteins, Cysteine-rich intestinal protein 1 (CRIP1) exhibited a marked increase in modification and played a central role in promoting synovial proliferation. Mechanistically, CRIP1 underwent MOF-mediated lactylation in RA synovial fibroblasts. Lactylated CRIP1 hijacked the cell-cycle regulator p21, disrupting its interaction with cyclin-dependent kinase 2 (CDK2), thereby facilitating the G1/S phase transition. Functionally, AAV-mediated delivery of a lactylation-deficient CRIP1 K49R significantly reduced synovial proliferation compared with WT CRIP1. Peptide-based interventions targeting CRIP1 K49 lactylation effectively inhibited synovial hyperplasia and disease severity in both Collagen II-induced arthritis (CIA) and humanized NSG chimeric models. Collectively, CRIP1 protein lactylation drives synovial proliferation in RA by hijacking p21 from CDK2, thereby facilitating cell cycle progression. Targeting this pathway may serve as a promising strategy for RA.
    Keywords:  Autoimmunity; Metabolism; Rheumatology
    DOI:  https://doi.org/10.1172/jci.insight.200928