In vivo base editing rescues primary hyperoxaluria type 1 in rats

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 editors which can efficiently introduce precisely targeted base alterations without double-strand DNA breaks.

Source: Kidney International - December 21, 2023 Category: Urology & Nephrology Authors: Zhoutong Chen, Dexin Zhang, Rui Zheng, Lei Yang, Yanan Huo, Dan Zhang, Xiaoliang Fang, Yueyan Li, Guofeng Xu, Dali Li, Hongquan Geng Tags: basic research Source Type: research