Stem Cell Therapies Benefit Alport Syndrome

doi:10.1681/ASN.2009010123

Stem Cell Therapies Benefit Alport Syndrome

2009-11-01

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DOI：10.1681/ASN.2009010123

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Patients with Alport syndrome progressively lose renal function as a result of defective type IV collagen in their glomerular basement membrane. In mice lacking the alpha 3 chain of type IV collagen (Col4A3 knockout mice), a model for Alport syndrome, transplantation of wild-type bone marrow repairs the renal disease. It is unknown whether cell-based therapies that do not require transplantation have similar potential. Here, infusion of wild-type bone marrow-derived cells into unconditioned, nonirradiated Col4A3 knockout mice during the late stage of disease significantly improved renal histology and function. Furthermore, transfusion of unfractionated wild-type blood into unconditioned, nonirradiated Col4A3 knockout mice improved the renal phenotype and significantly improved survival. Injection of mouse and human embryonic stem cells into Col4A3 knockout mice produced similar results. Regardless of treatment modality, the improvement in the architecture of the glomerular basement membrane is associated with die novo expression of the alpha 3(IV) chain. These data provide further support for testing cell-based therapies for Alport syndrome.

Keywords:

BASEMENT-MEMBRANES

MOUSE MODEL

IV COLLAGEN

INTERSTITIAL FIBROSIS

KIDNEY-DISEASE

RENAL-DISEASE

MICE

GLOMERULONEPHRITIS

ALPHA-3(IV)

SURVIVAL

Stem Cell Therapies Benefit Alport Syndrome

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