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U.S. scientists say they've used bone marrow-derived stem cells to reverse genetic kidney disease in mice.
Reporting in this week's Proceedings of the National Academy of Sciences, the team said the stem cells were able to regenerate damaged renal cells in a mouse model of Alport syndrome, the second most common genetic cause of kidney failure in humans.
Symptoms of Alport syndrome usually appear in childhood, and the disease typically results in end-stage renal failure by the time a patient reaches the teens, 20s or 30s.
The study offers the first example of how stem cells may prove useful in repairing defects and restoring organ function and also provides a potential new strategy for treating Alport syndrome.
"This is one of 31 human diseases that occur because of genetic defects in the body's extracellular matrix and basement membrane proteins," study senior author Raghu Kalluri, chief of the division of matrix biology at Beth Israel Deaconess Medical Center and associate professor of medicine at Harvard Medical School, explained in a prepared statement.
The extracellular matrix (ECM) is present throughout the body and is made of collagens, proteogylcans, noncollagenous glycoproteins and, in some tissues, elastin fibers. The ECM helps maintain the structural integrity of many tissues by constructing a "scaffold" for cells.
"In normal kidneys, a specialized form of extracellular matrix known as the glomerular basement membrane (GBM), composed primarily of type IV collagen, is the key component of the blood filtration apparatus," Kalluri said.
Genetic mutations in people with Alport syndrome cause structural damage to the GBM, which results in a breakdown of the kidneys' filtration system. There is no cure for Alport syndrome. Kidney transplantation or lifelong dialysis are the only treatment options.
About four weeks after the bone marrow-derived stem cells had been transplanted into the mice, about 10 percent of the cells had incorporated into damaged areas of the kidneys and emerged as healthy renal cells. This resulted in improvements in kidney function and repair to GBM damage.
More information
The U.S. National Library of Medicine has more about Alport syndrome.
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