Adeno-Associated Viral Vector (AAV)-Mediated In Vivo CRISPR-Cas9 Delivery.
This chapter detail a methodology using a rAAV vector carrying Staphylococcus aureus Cas9 (SaCas9) to target the murine Hao1 gene in liver parenchymal cells as a curative treatment for primary hyperoxaluria type 1 (PH1).
Andrea Llanos-Ardaiz, N. Zabaleta, Laura Torella et al.
· Methods in molecular biology · 0 citations