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Endothelial effects of 3-hydroxyglutaric acid: implications for glutaric aciduria type I
Chris Mühlhausen1, Nicola Ott, Fariba Chalajour
1Department of Pediatrics, University of Hamburg, Germany.
Insights
Glutaric aciduria type 1 (GA1) metabolite 3-hydroxyglutaric acid (3-OH-GA) impairs blood vessel function. This metabolite disrupts endothelial cell migration and vascular integrity, potentially explaining GA1-related vasculopathy.
Area of Science:
- Biochemistry
- Vascular Biology
- Metabolic Disorders
Background:
- Infants with Glutaric Aciduria Type 1 (GA1) exhibit intracranial vascular dysfunction.
- The specific metabolite 3-hydroxyglutaric acid (3-OH-GA) is implicated in GA1 pathogenesis.
Purpose of the Study:
- To investigate the effects of 3-OH-GA on endothelial cell function and vascular integrity.
- To elucidate the mechanisms underlying 3-OH-GA-induced vasculopathy in GA1.
Main Methods:
- In vitro studies using endothelial cells to assess migration, tube formation, and actin cytoskeleton.
- In vivo studies using chick chorioallantoic membrane assay to evaluate vascular responses to 3-OH-GA.
Main Results:
- 3-OH-GA inhibited basal and VEGF-induced endothelial cell migration and tube formation.
- 3-OH-GA treatment disrupted endothelial cell morphology, leading to Ve-cadherin loss and actin cytoskeleton disorganization.
- In vivo, 3-OH-GA induced vascular dilatation and hemorrhage, indicating impaired vascular integrity.
Conclusions:
- 3-OH-GA significantly impairs endothelial cell chemotaxis and compromises structural vascular integrity.
- These findings suggest 3-OH-GA plays a critical role in GA1-associated vasculopathic processes.
- Potential involvement of N-methyl-D-aspartate receptor-dependent and -independent pathways in GA1 pathogenesis is proposed.
Abstract:
Infants with glutaric aciduria type 1 (GA1) are subject to intracranial vascular dysfunction. Here, we demonstrate that the disease-specific metabolite 3-hydroxyglutaric acid (3-OH-GA) inhibits basal and vascular endothelial growth factor (VEGF)-induced endothelial cell migration. 3-OH-GA affects the morphology of VEGF-induced endothelial tubes in vitro because of partial disintegration of endothelial cells. These effects correlate with Ve-cadherin loss. Remarkably, 3-OH-GA treatment of human dermal microvascular endothelial cells leads to disruption of actin cytoskeleton. Local application of 3-OH-GA alone or in combination with VEGF in chick chorioallantoic membrane induces abnormal vascular dilatation and hemorrhage in vivo. The study demonstrates that 3-OH-GA reduces endothelial chemotaxis and disturbs structural vascular integrity in vitro and in vivo. These data may provide insight in the mechanisms of 3-OH-GA-induced vasculopathic processes and suggest N-methyl-D-aspartate receptor-dependent and -independent pathways in the pathogenesis of GA1.
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