PUBLICATION

ER-associated degradation pathway protein SEL1L plays an evolutionarily conserved role in platelet adhesion

Authors
Dahlgren, A.R., Careddu, F., Norris, J.W., Di Buduo, C.A., Stanger, L., Adili, R., Kropp, E.M., Li, Q., Holinstat, M., Biunno, I., Balduini, A., Tablin, F., Shavit, J.A., Finno, C.J.
ID
ZDB-PUB-260217-2
Date
2026
Source
The Journal of Clinical Investigation   136: (Journal)
Registered Authors
Shavit, Jordan
Keywords
Coagulation, Genetic diseases, Genetics, Hematology, Platelets, Vascular biology
MeSH Terms
  • Animals
  • Blood Platelets*/metabolism
  • Blood Platelets*/pathology
  • Endoplasmic Reticulum*/genetics
  • Endoplasmic Reticulum*/metabolism
  • Evolution, Molecular*
  • Horses
  • Humans
  • Megakaryocytes/metabolism
  • Megakaryocytes/pathology
  • Mice
  • Mice, Knockout
  • Mutation, Missense
  • Platelet Adhesiveness*
  • Proteins*/genetics
  • Proteins*/metabolism
  • Proteolysis
  • Thrombasthenia*/genetics
  • Thrombasthenia*/metabolism
  • Thrombasthenia*/veterinary
  • Zebrafish
PubMed
41697739 Full text @ Journal of Clin. Invest.
Abstract
SEL1L is a well-known protein in the ER-associated degradation (ERAD) pathway. While it is known to be expressed in platelets, SEL1L has never been shown to play an active role. Here, we present evidence that SEL1L regulates platelet function. We first identified SEL1L through the study of Atypical Equine Thrombasthenia (AET), an autosomal recessive platelet disorder found in thoroughbred horses. A missense variant in SEL1L (c.1810A>G p.Ile604Val) was found in AET-affected horses, which we show is associated with decreased protein expression. SEL1L is intracellular in equine platelets and localizes to the surface upon activation with thrombin. Platelets from homozygous horses exhibited substantially decreased spreading on immobilized collagen. Human megakaryocytes were found to have 2 SEL1L protein isoforms that increase in expression during megakaryopoiesis, although only 1 isoform was delivered to mature platelets. Studies using inducible mouse and constitutive zebrafish KOs demonstrated that SEL1L is necessary for efficient platelet or thrombocyte (fish equivalent) adhesion to sites of endothelial injury. These data reveal a previously undescribed and conserved role for the ERAD pathway in the etiology of AET and platelet function, and GWAS data suggest that it may play a role in human platelet disorders as well.
Genes / Markers
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Phenotype
Mutations / Transgenics
Human Disease / Model
Sequence Targeting Reagents
Fish
Antibodies
Orthology
Engineered Foreign Genes
Mapping