Article
In vivo base editing rescues primary hyperoxaluria type 1 in rats.
Kidney international - 1 Mar 2024
Chen Zhoutong, Zhang Dexin, Zheng Rui, Yang Lei, Huo Yanan, Zhang Dan, Fang Xiaoliang, Li Yueyan, Xu Guofeng, Li Dali, Geng Hongquan
Abstract excerpt
Primary hyperoxaluria type 1 (PH1) is a childhood-onset autosomal recessive disease, characterized by nephrocalcinosis, multiple recurrent urinary calcium oxalate stones, and a high risk of progressive kidney damage. PH1 is caused by inherent genetic defects of the alanine glyoxylate aminotransferase (AGXT) gene. The in vivo repair of disease-causing genes was exceedingly inefficient before the invention of base...
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