Department of Biochemistry, Jichi Medical University School of Medicine, Shimotsuke, Tochigi, Japan.
Methods Mol Biol. 2023;2637:195-211. doi: 10.1007/978-1-0716-3016-7_15.
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.
腺相关病毒 (AAV) 载体是将基因转移到肝脏的有效工具，已被用作治疗遗传性疾病的基因治疗药物。通过静脉注射携带由肝细胞特异性启动子驱动的目的基因的 AAV 载体，可以在肝实质细胞中高效表达靶基因。通过静脉内给药，AAV 载体可以递送基因组编辑工具，包括 Cas9 和 gRNA，从而有效地在体内破坏肝脏中的靶基因表达。通过给予 AAV 载体，我们可以快速获得肝脏中特定基因表达缺失的小鼠。该方法可能适用于开发遗传性疾病的基因组编辑治疗方法，以及探索来自肝实质细胞的靶基因的生理作用的基础研究。
