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. Overall design: 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（AAV）变体对人肝细胞与小鼠肝细胞，或门静脉周围肝细胞与中央静脉周围肝细胞的嗜性可能存在差异。本研究采用单核RNA测序（single nuclear RNA-seq）技术，检测了人肝细胞在FRG小鼠模型中的植入情况，以及该模型中肝细胞的转导效果。
整体实验设计：植入FRG小鼠肝脏的人肝细胞的单核RNA测序样本，包括植入前、植入后5个月的样本，以及经AAV处理的样本。