腺相关病毒（Adeno-associated virus，AAV） 是目前发现结构最简单的、无包膜的单链DNA病毒，病毒基因组长约有4.7Kb，属于细小病毒家族。 AAV自身不能复制，必须依赖于其它病毒复制，比如腺病毒、疱疹病毒、杆状病毒，有超过80%的人携带AAV，未发现AAV与任何疾病相关。 重组腺相关病毒 （Recombinant AAV，rAAV） 是经工程改造的AAV载体，它剔除了AAV基因组全部编码蛋白Rep或Cap基因序列，只在两端保留作为包装信号的反式DNA序列（即ITR）。 …

2024年4月3日 · Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term...

目前在临床试验中使用的aav载体少于12种血清型，其中使用频率最高的是aav2。值得注意的是，更新和更有效的衣壳，如aav8、aav9和aavrh.10正在用于越来越多的试验。

2023年3月14日 · Combining synthetic, molecular, and chemical biology, we have created a novel class of programmable AAV vectors, Nε-AAVs, by utilizing single unnatural amino acid insertion. We characterized different mutant capsids of Nε-AAV vectors and successfully conjugated them by click chemistry.

2014年1月1日 · In this study, we have investigated the potential of three cell-permeable peptides (Antp, TAT-HA2, and LAH4) to enhance AAV2-mediated gene delivery. We found that these CPPs significantly increase AAV2-mediated transduction into tissues and cells, including both permissive and nonpermissive cells.

In this review, we focus on AAV vector-based gene therapy, shedding light on this promising technique and its remarkable success in haemophilia, with a special focus on hepatic complications and their management in daily clinical practice.

2022年7月15日 · Here, we examined a potential therapeutic application of AAV-mediated hepatic expression of the oxidized phospholipid-binding antibody fragment scFv-E06 for the prevention of the initiation of NAFLD and, independently, of the progression to NASH and hepatic fibrosis.

2021年12月3日 · Schaffer and colleagues implemented a genome-wide activation screen to identify host cell target genes that promote AAV production. In particular, SKA2 and ITPRIP upregulation significantly increased AAV production, due to their roles in promoting vector genome replication and the AAV full/empty capsid ratio.

2019年9月30日 · To address this discrepancy humanized mouse models have recently been used to predict AAV transduction efficiency for human hepatocytes. In this study we evaluated AAV vector transduction from...

2009年3月15日 · Adeno-associated virus serotype 2 (AAV-2) is a leading candidate vector for gene therapy. Cell entry starts with attachment to a primary receptor, Heparan Sulfate Proteoglycan (HSPG) before binding to a co-receptor. Here, cryo-electron microscopy provides direct visualization of the virus–HSPG interactions.