HAX1, gene responsible for Kostmann syndrome, regulates gingival epithelial barrier function via intracellular trafficking of JAM1

BackgroundKostmann syndrome is an autosomal recessive disorder caused by a mutation of the hematopoietic cell-specific Lyn substrate 1 associated protein X-1 (HAX1) gene, and characterized by low number of neutrophils and increased susceptibility to infections. Additionally, Kostmann syndrome is kno...

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Main Authors: Keita Tanigaki, Tsukasa Tamamori, Naoko Sasaki, Risako Matsumura, Shunsuke Yamaga, Akito Sakanaka, Atsuo Amano, Michiya Matsusaki, Hiroki Takeuchi, Masae Kuboniwa
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-08-01
Series:Frontiers in Cell and Developmental Biology
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Online Access:https://www.frontiersin.org/articles/10.3389/fcell.2025.1624718/full
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author Keita Tanigaki
Tsukasa Tamamori
Naoko Sasaki
Risako Matsumura
Shunsuke Yamaga
Akito Sakanaka
Atsuo Amano
Michiya Matsusaki
Hiroki Takeuchi
Masae Kuboniwa
author_facet Keita Tanigaki
Tsukasa Tamamori
Naoko Sasaki
Risako Matsumura
Shunsuke Yamaga
Akito Sakanaka
Atsuo Amano
Michiya Matsusaki
Hiroki Takeuchi
Masae Kuboniwa
author_sort Keita Tanigaki
collection DOAJ
description BackgroundKostmann syndrome is an autosomal recessive disorder caused by a mutation of the hematopoietic cell-specific Lyn substrate 1 associated protein X-1 (HAX1) gene, and characterized by low number of neutrophils and increased susceptibility to infections. Additionally, Kostmann syndrome is known to be complicated by periodontitis, though the etiological molecular basis remains unclear. We previously reported findings showing that junctional adhesion molecule 1 (JAM1), a tight junction-associated protein, has an important role to maintain epithelial barrier function in gingival tissues, which prevents penetration of bacterial virulence factors, such as lipopolysaccharide (LPS) and peptidoglycan (PGN). In the present study, the effects of HAX1 on gingival barrier function were investigated.ResultsExaminations of immortalized human gingival epithelial (IHGE) cells showed HAX1 localization in mitochondria. In HAX1-knockdown IHGE cells, significantly decreased levels of JAM1 were found. Additionally, cisplatin, a chemotherapeutic agent reported to inhibit HAX1, also led to decreased expression of both HAX1 and JAM1. Furthermore, JAM1 was scarcely detected in HAX1-knockout cells, while administration of bafilomycin A1, a lysosomal inhibitor, restored JAM1 expression in those cells. Finally, using a three-dimensional multilayered gingival epithelial tissue model, HAX1 knockout along with cisplatin administration was also found to increase permeability to LPS and PGN, which was dependent on JAM1 expression.ConclusionThese results indicate that periodontal diseases complicated with Kostmann syndrome are induced by reduced JAM1 expression, caused by JAM1 being missorted into lysosomes by HAX1 dysfunction.
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spelling doaj-art-6157afee8c344589aeab4e736a962a6d2025-08-20T03:37:31ZengFrontiers Media S.A.Frontiers in Cell and Developmental Biology2296-634X2025-08-011310.3389/fcell.2025.16247181624718HAX1, gene responsible for Kostmann syndrome, regulates gingival epithelial barrier function via intracellular trafficking of JAM1Keita Tanigaki0Tsukasa Tamamori1Naoko Sasaki2Risako Matsumura3Shunsuke Yamaga4Akito Sakanaka5Atsuo Amano6Michiya Matsusaki7Hiroki Takeuchi8Masae Kuboniwa9Department of Preventive Dentistry, Graduate School of Dentistry, The Osaka University, Osaka, JapanDepartment of Preventive Dentistry, Graduate School of Dentistry, The Osaka University, Osaka, JapanJoint Research Laboratory (TOPPAN) for Advanced Cell Regulatory Chemistry, Graduate School of Engineering, The Osaka University, Osaka, JapanDepartment of Preventive Dentistry, Graduate School of Dentistry, The Osaka University, Osaka, JapanDepartment of Preventive Dentistry, The Osaka University Dental Hospital, Osaka, JapanDepartment of Preventive Dentistry, Graduate School of Dentistry, The Osaka University, Osaka, JapanDepartment of Preventive Dentistry, Graduate School of Dentistry, The Osaka University, Osaka, JapanDepartment of Applied Chemistry, Graduate School of Engineering, The Osaka University, Osaka, JapanDepartment of Preventive Dentistry, The Osaka University Dental Hospital, Osaka, JapanDepartment of Preventive Dentistry, Graduate School of Dentistry, The Osaka University, Osaka, JapanBackgroundKostmann syndrome is an autosomal recessive disorder caused by a mutation of the hematopoietic cell-specific Lyn substrate 1 associated protein X-1 (HAX1) gene, and characterized by low number of neutrophils and increased susceptibility to infections. Additionally, Kostmann syndrome is known to be complicated by periodontitis, though the etiological molecular basis remains unclear. We previously reported findings showing that junctional adhesion molecule 1 (JAM1), a tight junction-associated protein, has an important role to maintain epithelial barrier function in gingival tissues, which prevents penetration of bacterial virulence factors, such as lipopolysaccharide (LPS) and peptidoglycan (PGN). In the present study, the effects of HAX1 on gingival barrier function were investigated.ResultsExaminations of immortalized human gingival epithelial (IHGE) cells showed HAX1 localization in mitochondria. In HAX1-knockdown IHGE cells, significantly decreased levels of JAM1 were found. Additionally, cisplatin, a chemotherapeutic agent reported to inhibit HAX1, also led to decreased expression of both HAX1 and JAM1. Furthermore, JAM1 was scarcely detected in HAX1-knockout cells, while administration of bafilomycin A1, a lysosomal inhibitor, restored JAM1 expression in those cells. Finally, using a three-dimensional multilayered gingival epithelial tissue model, HAX1 knockout along with cisplatin administration was also found to increase permeability to LPS and PGN, which was dependent on JAM1 expression.ConclusionThese results indicate that periodontal diseases complicated with Kostmann syndrome are induced by reduced JAM1 expression, caused by JAM1 being missorted into lysosomes by HAX1 dysfunction.https://www.frontiersin.org/articles/10.3389/fcell.2025.1624718/fullHAX1JAM1Kostmann syndromecisplatinperiodontitisbarrier
spellingShingle Keita Tanigaki
Tsukasa Tamamori
Naoko Sasaki
Risako Matsumura
Shunsuke Yamaga
Akito Sakanaka
Atsuo Amano
Michiya Matsusaki
Hiroki Takeuchi
Masae Kuboniwa
HAX1, gene responsible for Kostmann syndrome, regulates gingival epithelial barrier function via intracellular trafficking of JAM1
Frontiers in Cell and Developmental Biology
HAX1
JAM1
Kostmann syndrome
cisplatin
periodontitis
barrier
title HAX1, gene responsible for Kostmann syndrome, regulates gingival epithelial barrier function via intracellular trafficking of JAM1
title_full HAX1, gene responsible for Kostmann syndrome, regulates gingival epithelial barrier function via intracellular trafficking of JAM1
title_fullStr HAX1, gene responsible for Kostmann syndrome, regulates gingival epithelial barrier function via intracellular trafficking of JAM1
title_full_unstemmed HAX1, gene responsible for Kostmann syndrome, regulates gingival epithelial barrier function via intracellular trafficking of JAM1
title_short HAX1, gene responsible for Kostmann syndrome, regulates gingival epithelial barrier function via intracellular trafficking of JAM1
title_sort hax1 gene responsible for kostmann syndrome regulates gingival epithelial barrier function via intracellular trafficking of jam1
topic HAX1
JAM1
Kostmann syndrome
cisplatin
periodontitis
barrier
url https://www.frontiersin.org/articles/10.3389/fcell.2025.1624718/full
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