Targeted sequencing of Hao1 mouse gene treated with paired D10ASaCas9 nickases
This study aimed to permanently reduce GO (Hao1 gene) expression by the use of paired Staphylococcus aureus Cas9 nickases (D10ASaCas9) targeting the Hao1 that was delivered to Primary hyperoxaluria type 1 (PH1) model mice using AAV vectors. The therapeutic aim was to produce a significant decrease in GO expression by creating indels and a reduction in oxalate overproduction.