Adenine base editing rescues pathogenic phenotypes in tissue engineered vascular model of Hutchinson-Gilford progeria syndrome.

The rare, accelerated aging disease Hutchinson-Gilford Progeria Syndrome (HGPS) is commonly caused by a c.1824 C > T point mutation of the gene that results in the protein progerin. The primary cause of death is a heart attack or stroke arising from atherosclerosis.

Development and Application of Endothelial Cells Derived From Pluripotent Stem Cells in Microphysiological Systems Models.

The vascular endothelium is present in all organs and blood vessels, facilitates the exchange of nutrients and waste throughout different organ systems in the body, and sets the tone for healthy vessel function. Mechanosensitive in nature, the endothelium responds to the magnitude and temporal waveform of shear stress in the vessels. Endothelial dysfunction can lead to atherosclerosis and other diseases.