MEK Inhibition Reduces Vascular Malformations and Gene Dysregulation in <i>NRAS</i> <sup>Q61R</sup> Human Endothelial Cells
Trametinib was the most effective MEK inhibitor, correcting some dysregulated genes in NRAS<sup>Q61R</sup> ECs, including some in the Notch pathway. Trametinib reduced vessel overgrowth in xenografts of NRAS<sup>Q61R</sup> ECs. These studies support using trametinib treatment for KLA patients with an NRAS<sup>Q61R</sup> mutation.
Authors
- Andrew J. Wagner (ORCID: https://orcid.org/0000-0002-4384-9448)
- Sara Alharbi (ORCID: https://orcid.org/0000-0001-9889-7474)
- Svatava Merkle (ORCID: https://orcid.org/0000-0001-7604-3849)
- Patricia Pastura
- Yan Xu (ORCID: https://orcid.org/0000-0002-2832-2664)
- Dermot Fox
- Timothy D. Le Cras
- C. Griffin McDaniel
Institutions
- Cincinnati Children's Hospital Medical Center (US)
- University of Cincinnati Medical Center (US)
Publication Details
- Journal
- Pediatric Blood & Cancer
- Published
- 2026-01-07
- DOI
- https://doi.org/10.1002/1545-5017.70002
- Primary Topic
- Vascular Malformations and Hemangiomas
- Type
- article
- Field-Weighted Citation Impact
- 0.00