Alport syndrome is a progressive hereditary kidney disease with no definitive therapy. It is caused by mutations in any of the collagen IV genes (COL4A3, COL4A4, and COL4A5).
Motoko Yanagita and colleagues, at Kyoto University Graduate School of Medicine, Japan, have now identified a role for the protein USAG-1 in the development of disease in mice that model Alport syndrome (Col4a3-/- mice).
As deletion of Usag1 in Col4a3-/- mice led to substantial attenuation of disease progression, preservation of kidney function, and extension of life span, the authors suggest that inhibiting USAG-1 might be a promising therapeutic approach for the treatment of Alport syndrome.
The research appears in the Journal of Clinical Investigation.
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