PIEZO1 as a potential therapeutic target in Marfan syndrome

Graphical AbstractMarfan syndrome (MFS) is caused by mutations of the fibrillin-1 gene, which compromise extracellular matrix integrity and activate local transforming growth factor-β (TGF-β) signalling, leading to formation of a thoracic aortic aneurysm (TAA). Expression of the mechanosensitive ion channel PIEZO1 is reduced in the aorta of MFS mice, leading to formation of a TAA by inhibiting endocytosis and autophagy of TGF-β receptor 2 (TGFBR2) through Rab GTPase 3C (RAB3C). Activation of PIEZO1 by Yoda1 inhibits TAA formation through its superior multifaceted effect on MFS. Created in BioRender. Komuro, J. (2024) https://BioRender.com/b78s839.Open in new tabDownload slide