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Constitutively active Lyn kinase causes a cutaneous small vessel vasculitis and liver fibrosis syndrome

de Jesus, A. A.; Yang, D.; Chen, G.; Brdicka, T.; Ruth, N.; Bennin, D.; Cebecauerova, D.; Malcova, H.; Freeman, H.; Martin, N.; Svojgr, K.; Passo, M.; Bhuyan, F.; Alehashemi, S.; Rastegar, A.; Uss, K.; Kardova, L.; Marrero, B.; Duric, I.; Omoyinmi, E.; Peldova, P.; Lee, C.-C. R.; Kleiner, D. E.; Hadigan, C. M.; Hewitt, S. M.; Pittaluga, S.; Carmona-Rivera, C.; Calvo, K. R.; Shah, N.; Balascakova, M.; Fink, D.; Kotalova, R.; Parackova, Z.; Peterkova, L.; Kuzilkova, D.; Campr, V.; Sramkova, L.; Biancotto, A.; Brooks, S. R.; Manes, C.; Meffre, E.; Harper, R. L.; Kuehn, H.; Kaplan, M. J.; Brogan,

2022-09-29 genetic and genomic medicine
10.1101/2022.09.27.22280319 medRxiv
Show abstract

Neutrophilic inflammation is a hallmark of many monogenic autoinflammatory diseases; pathomechanisms that regulate extravasation of damaging immune cells into surrounding tissues are poorly understood. We identified three unrelated boys with perinatal-onset of neutrophilic cutaneous small vessel vasculitis and systemic inflammation. Two patients developed liver fibrosis in their first year of life. Next-generation sequencing identified two de novo truncating variants in the Src-family tyrosine kinase, LYN, p.Y508*, p.Q507* and a de novo missense variant, p.Y508F, that result in constitutive activation of Lyn kinase. Functional studies reveal increased expression of ICAM-1 on induced patient-derived endothelial cells (iECs) and of {beta}2-integrins on neutrophils that increase neutrophil adhesion and vascular transendothelial migration (TEM). Treatment with TNF inhibition improved systemic inflammation; and liver fibrosis resolved on treatment with the Src kinase inhibitor dasatinib. These findings reveal a critical role for Lyn kinase in modulating inflammatory signals, regulating microvascular permeability and neutrophil recruitment, and in promoting hepatic fibrosis.

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