Several cell-based therapy approaches could provide new treatments for patients with Alport syndrome, reports an upcoming paper in the Journal of the American Society of Nephrology (JASN). "Our study opens up many considerations of how new therapies related to the use of stem cells can be devised for our kidney patients with chronic disease," comments Raghu Kalluri, MD, PhD (Harvard Medical School, Boston, MA). Led by Valerie LeBleu, PhD (also of Harvard Medical School), the researchers tested various types of cell-based therapy in mice with a gene defect similar to that causing Alport syndrome, a genetic kidney disease. Most often occurring in boys, Alport syndrome causes progressive kidney disease leading to kidney failure at a young age. Patients may develop hearing loss and eye disease as well. Although treatment can slow the progression of kidney disease, there is currently no cure for Alport syndrome. The experiments provide evidence that stem cell treatments could repair the kidney defects associated with Alport syndrome. "We found that stem cells derived from adult bone marrow are equally useful as embryonic stem cells," says Kalluri. "This will make it easier to translate these scientific discoveries to a treatment protocol for patients with Alport syndrome."
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