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Cell-Based Therapies May Be Beneficial in Alport Syndrome

Last Updated: October 16, 2009.

Cell-based therapies may offer hope to patients with Alport syndrome, according to an animal study published online Oct. 15 in the Journal of the American Society of Nephrology.

FRIDAY, Oct. 16 (HealthDay News) -- Cell-based therapies may offer hope to patients with Alport syndrome, according to an animal study published online Oct. 15 in the Journal of the American Society of Nephrology.

In mice with a model of Alport syndrome, Valerie LeBleu, Ph.D., of the Beth Israel Deaconess Medical Center and Harvard Medical School in Boston, and colleagues infused wild-type bone marrow-derived cells, transfused unfractionated wild-type blood, or injected mouse and human embryonic stem cells during the late stage of the disease in mice lacking the α3 chain of type IV collagen (Col4A3 knockout mice).

The researchers found that these approaches rescued the renal disease without any need for total body irradiation, and improved the renal phenotype, survival, and function by enabling the production and incorporation of the missing type IV collagen.

"Our work supports the development of a cell-based therapy for patients with Alport syndrome," the authors conclude. "Currently, therapies that can dramatically change the disease progression in patients with Alport syndrome are lacking. The experiments highlighted in this report offer hope for future potential cell-based therapy for our patients."

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