Research ArticleCARDIOMYOPATHY

Clinical trial in a dish using iPSCs shows lovastatin improves endothelial dysfunction and cellular cross-talk in LMNA cardiomyopathy

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Science Translational Medicine  29 Jul 2020:
Vol. 12, Issue 554, eaax9276
DOI: 10.1126/scitranslmed.aax9276

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Lovastatin for cardiolaminopathy

Mutations in LMNA, which encodes nuclear envelope proteins, can cause dilated cardiomyopathy and vascular endothelial dysfunction. To understand mechanisms contributing to this disease, Sayed et al. studied induced pluripotent stem cell–derived endothelial cells (iPSC-ECs) from a family harboring an LMNA mutation. They found down-regulation of KLF2, a protein involved in mechanotransduction, which caused endothelial dysfunction. Lovastatin could induce KLF2 in iPSC-ECs, improving cardiomyocyte function in coculture and clinical EC function in two patients treated with the drug. This study demonstrates a workflow for identifying and validating potential drug treatments for patients with cardiolaminopathy.

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