近日,MIT和哈佛大学Broad研究所的研究人员在 Nature 子刊Nature Communications上发表了题为:Systematic multi-trait AAV capsid engineering for efficient gene delivery的研究论文。 该研究开发了一种用于系统性设计多特征AAV衣壳的通用机器学习方...
AAV capsid tropism refers to the ability of the viral capsid to selectively infect and transduce specific cell types based on the presence of specific surface receptors or other factors on the target cells.AAV capsid engineering is an important approach in therapies for cancer or genetic disorder ...
随着基因治疗的蓬勃发展，以AAV为载体的基因疗法在许多疾病中显示出突破性的疗效，甚至具有完全治愈疾病的潜力。虽然也存在自身的局限性和一定的安全性风险，但它仍是基因治疗的主流选择。AAV属于微小型，非致病性病毒，具有大约20-26 nm的无包膜二十面体衣壳。主要由两部分组成：蛋白衣壳（Capsid）和包裹在衣壳内的...
[2] Li C, Samulski R J. Engineering adeno-associated virus vectors for gene therapy[J]. Nature Reviews Genetics, 2020, 21(4): 255-272. [3] Lundgren T S, Denning G, Stowell S R, et al. Pharmacokinetic analysis identifies a fac...
[2] L.Zhang,et al.Capsid Engineering Overcomes Barriers Toward Adeno-Associated Virus Vector-Mediated Transduction of Endothelial Cells. Hum Gene Ther. 2019 Sep;30(10):1-46. doi:10.1089/hum.2019.027. [3] Krolak T,et al. A High-Efficiency AAV for Endothelial Cell Transduction Throughout the...
capsid engineeringcapsid shufflinghepatocyte transductionliver-directed gene therapysite-directed mutagenesisIntroduction: Gene therapy clinical trials with adeno-associated virus (AAV) vectors report impressive clinical efficacy data. Nevertheless, challenges have become apparent, such as the need for high ...
[2] AAV manufacturing for clinical use: insights on current challenges from the upstream process perspective[J]. Current Opinion in Biomedical Engineering, 2021, 20: 100353.[3] Adeno-associated virus (AAV) assembly-activating protein is not an essential requirement for capsid assembly of AAV ...
AAV capsid engineering, especially direct evolution engineering, can generate highly specific serotypes that target muscle and/or heart [3]. However, new capsid engineering is a laborious process of screening millions of capsid variants before the desired specificity is met [3]. This is a multi-...
5.Zhang L, Rossi A, Lange L, et al. Capsid Engineering Overcomes Barriers Toward Adeno-...
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