- Patients with progeria develop a disease of the blood vessels which contributes to their early death
- Scientists have developed a 3D model of blood vessels that resemble the diseased blood vessels of progeria
- The model can be used to screen medications for the treatment of progeria
Scientists have developed blood vessels in the laboratory that are
similar to the blood vessels of progeria patients. These can serve as a model
to screen drugs that could possibly be useful in patients with the rare genetic
disorder. The research was published in the Scientific Reports.
Hutchison-Gilford Progeria Syndrome (HGPS) is an extremely rare genetic disorder where the affected person shows accelerated aging - a young child appears like an old person. The lifespan of these individuals is short - usually less than 14 years. Affected individuals usually die of cardiovascular disease.
Progeria results due to the accumulation of a protein called progerin within cells. Changes noted in the smooth muscle layer of blood vessels, which is the middle layer of the blood vessels, include excess calcium and fat deposits, thickening of the blood vessel layer and fibrosis. It results in atherosclerosis or stroke, which results in early death.
The smooth muscle cells for the model were produced from human induced pluripotent stem cell (iPSC) obtained from a progeria patient. The endothelial cell layer, the inner cellular lining of the blood vessel, was derived from cells from a healthy donor. The blood vessels showed properties similar to those of a progeria patient after four weeks, which included:
- Reduced vasoactivity
- Increased thickness of the middle muscular layer
- Increased calcification and apoptosis
These findings were as compared to a similar model made from cells of a patient without progeria.
The scientists also tested the drug everolimus on the vascular model. Everolimus has been suggested to provide some benefit in progeria, based on previous testing on 2D cell cultures. When everolimus was tested on the vascular model, it improved the function and increased the expression of smooth muscle contractile proteins. It, however, did not improve the calcification.
The vascular model thus appears to have a promising role in the screening of new drugs for progeria. It could also prove to be useful to study other cardiovascular diseases associated with aging that brings about similar changes in the blood vessels. Several issues have yet to be addressed, for example, the vessels do not show accumulation of lipids, in contrast to what is noted in progeria patients. The model contains only smooth muscle cells from progeria patients and not the endothelial cells. The system is also not connected to any organ or exposed to the complexities of the human system.
- Atchison L, Zhang H, Cao K, Truskey GA. A Tissue Engineered Blood Vessel Model of Hutchinson-Gilford Progeria Syndrome Using Human iPSC-derived Smooth Muscle Cells. Scientific Reports, Aug. 15, (2017). DOI:10.1038/s41598-017-08632-4
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