Lab-grown blood vessels provide platform for studying premature aging syndrome and other rare diseases
Biomedical engineers have grown miniature human blood vessels that exhibit many of the symptoms and drug reactions associated with Hutchinson-Gilford Progeria Syndrome—an extremely rare genetic disease that causes symptoms resembling accelerated aging in children.
The technology will help doctors and researchers screen potential therapeutics for the disease more rapidly, with the goal of eventually creating a platform for personalized screening. The technique also offers a new way to study other rare diseases and could provide insights into treating heart disease in the elderly.
The study was published online on August 15 in the journal Scientific Reports.
“One of the drugs currently prescribed for this disease extends patients’ lives by three months, and that’s been considered a major feat,” said Leigh Atchison, a doctoral candidate in biomedical engineering at Duke University and first author of the study. “They’re looking for anything that will extend lifespan by even a few months. It’s that devastating.”
Hutchinson-Gilford Progeria Syndrome—or simply progeria for short—is a non-hereditary genetic disease caused by a single-point mutation in the genome. It is so rare and so deadly that there are currently only about 250 known cases worldwide.
Progeria is triggered by a defective protein called progerin that accumulates outside of a cell’s nucleus rather than becoming part of its structural support system. This causes the nucleus to take on an abnormal shape and inhibits its ability to divide. The resulting symptoms look much like accelerated aging, and affected patients usually die of heart disease brought on by weakened blood vessels before the age of 14.
“Progeria isn’t considered hereditary, because nobody lives long enough to pass it on,” said George Truskey, the R. Eugene and Susie E. Goodson Professor of Biomedical Engineering…