Stringent selection of AAV variants in vivo using FRG mice partially repopulated with primary human hepatocytes results in AAV-LK03 (Ref. 12). In comparison to AAV-DJ, which efficiently transduces most mouse and human cells in culture, AAV …

Plasmid AAV-LK03 from Dr. Mark Kay's lab contains the insert LK03 capsid and is published in Nature. 2014 Feb 20;506(7488):382-6. doi: 10.1038/nature12875. Epub 2013 Dec 25. This plasmid is available through Addgene.

2023年3月9日 · De-targeting AAV-LK03 from HSPG yielded a vector that consistently transduced a larger area of the chimeric liver in hFRG, as validated by complementary strategies including NGS (Figure 5), immunofluorescence (Figure 6), and sn-RNA-seq (Figure 7).

Stringent selection of AAV variants in vivo using FRG mice partially repopulated with primary human hepatocytes results in AAV-LK03. In comparison to AAV-DJ, which efficiently transduces most mouse and human cells in culture, AAV-LK03 preferentially transduces human cells.

研究团队选取了aav8、aav5、aav-lk03、aav6和aav-np59这五种常见或新兴的aav血清型，将其以共注射的方式分别注入正常和脂肪变性的离体人肝脏组织。

We found a chimeric capsid (LK03) that after vectorization (rAAVLK03) was 10 to 20 times more efficient at transducing human hepatocytes in vivo compared to rAAV8 with the same expression cassette. 4 Although the LK03 capsid most closely resembles AAV3B, based on total amino acid sequence, this capsid is derived from at least four different ...

Stanford inventors have engineered an adeno-associated virus (AAV) variant on the existing LK03 platform that enables this highly efficient primate-specific serotype for use in rodent preclinical studies.

AAV-LK03的高效转导：在体外实验中，AAV-LK03是人类足细胞的高效转导剂。 功能恢复：AAV-LK03介导的足细胞蛋白在突变人类足细胞中实现了功能恢复。 预防性策略的效果：在诱导型Podocin敲除小鼠模型中，AAV治疗组小鼠的尿白蛋白/肌酐比 (ACR)、血清肌酐和尿素水平显著降低，血清白蛋白水平有所升高。

We used a capsid-shuffled rAAV library to perform directed evolution in human iPSC-derived cardiomyocytes (hiPSC-CMs). Five candidates emerged, with four presenting high sequence identity to AAV6,...