Genome Editing of Murine Liver Hepatocytes by AAV Vector-Mediated Expression of Cas9 In Vivo.

Adeno-associated virus (AAV) vectors are attractive tools for gene transfer to the liver and are used as gene therapeutic drugs for inherited disorders. The intravenous injection of an AAV vector harboring the gene of interest driven by the hepatocyte-specific promoter could efficiently express the target gene in liver hepatocytes. The delivery of genome editing tools including Cas9 and gRNA, by the AAV vector, can efficiently disrupt the target gene expression in the liver in vivo by intravenous administration in mice. We can quickly obtain mice lacking specific gene expression in the liver only by administering the AAV vector. The method could be suitable for developing genome editing treatments for inherited disorders and basic research exploring the physiological role of the target gene produced from liver hepatocytes.