Publications by authors named "Anna L McCarter"
Trametinib inhibits lymphatic vessel invasion of bone in a mouse model of Gorham-Stout disease.
J Vasc Anom (Phila)
December 2023
Objective: Gorham-Stout disease (GSD) is a rare lymphatic anomaly that can be caused by somatic activating mutations in . This discovery has led investigators to suggest that MEK inhibitors could be a novel treatment for GSD. However, the effect of MEK inhibitors on bone disease in animal models of GSD has not been investigated.
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- Gorham-Stout disease (GSD) is a rare lymphatic disorder causing abnormal lymphatic growth in bones, leading to severe complications like chylothorax, with unclear causes and insufficient treatments.
- Researchers discovered a specific mutation in the KRAS gene linked to GSD and created a mouse model (iLECKras) to study its effects, finding that it caused bone lymphatics and abnormal lymphatic valve development.
- The study also tested trametinib, a drug that inhibited a signaling pathway related to KRAS, showing promise in mitigating symptoms in the mouse model, suggesting potential for treating GSD in patients.
Bones do not normally have lymphatics. However, individuals with generalized lymphatic anomaly (GLA) or Gorham-Stout disease (GSD) develop ectopic lymphatics in bone. Despite growing interest in the development of tissue-specific lymphatics, the cellular origin of bone lymphatic endothelial cells (bLECs) is not known and the development of bone lymphatics has not been fully characterized.
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