Development of an AAV-LK03 variant with improved liver lobular biodistribution in the humanised FRG model II

AAV variants may differ in their tropism for human vs mouse hepatocytes or portal vs central hepatocytes. We examined the engraftment of human hepatocytes in the FRG mouse model and the transduction of hepatocytes in this model using single nuclear RNA-seq. Single nuclear RNA-seq of human hepatocytes engrafted into FRG mouse liver, including pre- and 5 months post-engrafted, as well as AAV-treated samples

腺相关病毒（Adeno-associated virus, AAV）变体对人源肝细胞与小鼠肝细胞，或门静脉区肝细胞与中央区肝细胞的嗜性或存在差异。本研究采用单核RNA测序（single nuclear RNA-seq）技术，检测了人源肝细胞在FRG小鼠模型中的嵌合定植情况，以及该模型中肝细胞的转导过程。本研究针对植入FRG小鼠肝脏的人源肝细胞开展了单核RNA测序，样本涵盖定植前、定植后5个月的样本，以及经AAV处理的样品。