When recombinant AAV (rAAV) is used for research purposes, the Rep protein is supplied in trans, eliminating the ability of rAAV to integrate into its preferred site of genomic integration on human chromosome 19, termed AAVS1. Instead, the rAAV genome is typically processed into a double-stranded circular episome through double stranded synthesis.

2022年6月22日 · 目前比较流行的一种重组AAV载体制备方法是三种质粒瞬转进入组成型表达腺病毒E1a和E1b基因的HEK293细胞，其中一个质粒表达腺相关病毒的rep和cap基因；另一个质粒表达需要在体内递送的目标基因；还有一个质粒表达具有辅助功能的腺病毒基因，例如表达分别负责 ...

rep基因编码病毒复制所需的四种蛋白质；它们以其分子量命名：Rep78、Rep68、Rep52和Rep40。 cap基因 通过来自不同起始密码子的可变剪接和翻译编码三个衣壳亚基。

5 天之前 · Recombinant adeno-associated virus (AAV) is a prominent gene therapy vector, offering a strong safety profile and sustained gene expression. ... Rep/Cap, and transgene into three plasmids, or a dual-plasmid system, which combines Rep, Cap, and helper genes into one plasmid along with a separate transgene plasmid.

腺相关病毒（Adeno-associated virus，AAV）属于细小病毒科依赖病毒属，是目前发现的一类结构简单、无包膜的单链DNA缺陷型病毒，它的生命周期依赖于复制病毒的参与，如腺病毒和单纯疱疹病毒。

2024年3月27日 · To gain a comprehensive understanding of the Rep proteins and their mutational landscape, we assayed the effects of all 39,297 possible single-codon mutations to the AAV2 rep gene on AAV2 production. Most beneficial variants are not observed in nature, indicating that improved production may require synthetic mutations.

本文主要关注了 adeno-associated virus (AAV) 的Rep蛋白的结构和功能。研究者们通过创建AAV2 rep基因的全面突变库，对所有可能的单密码子替换和缺失变体进行了深入的探索。

Infectious rep-negative AAV progeny was produced at varying efficiencies depending on the rep expression construct used. These data show that functional expression of full-length Rep in recombinant cell lines is possible and that the state of Rep expression is critical for the infectivity of AAV progeny produced. These references are in PubMed.

2025年3月20日 · e, Representative images of AAV concatemers detected with SpECTr in PCs of dual-injected WT and SCID animals shown in Fig. 4a. HCR-FISH against Itpr1 transcript serves as a marker for PCs. Scale ...

重组腺相关病毒 (Recombinant adeno-associated viruses,rAAVs)是主要的 基因治疗载体。 几种rAAV载体疗法已获得监管部门的批准，但生产足够数量的rAAV仍然很困难。 AAV Rep蛋白 是基因组复制和包装所必需的，是有望提升rAAV产量的工程靶标，但仍未得到充分的研究。