pAAVone-AAV3B-CMV-EGFP-2.8kb

pAAVone-AAV3B-CMV-EGFP-2.8kb is a plasmid used to produce AAV3B Reference (Full) and AAV3B Reference (Byproduct-Empty) using novel AAVone production system. It integrates all the AAV production necessary components, including Ad helper genes (E2A, E4orf6 and VA RNA), AAV helper genes (Rep and AAV3B), and a 2.8kb single-strand AAV-CMV-EGFP genome. AAV3B-CMV-EGFP viruses can be simply and efficiently produced by transfection of this single-into host cells, such us HEK 293 and HEK 293T. AAV3B is a serotype of adeno-associated virus that has shown promising potential for liver-targeted gene therapy applications. Compared to other AAV serotypes, AAV3B has a unique tissue tropism, with higher transduction efficiency in human hepatocytes. This makes it an attractive option for treating liver-specific genetic diseases such as hemophilia or metabolic disorders.

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Background

Our data demonstrated that AAV3b exhibits low RNA expression in the mouse (C57B6) tissues, but may have related high DNA level in kidney after IV injection.pAAVone plasmids are used to produce AAVs using novel AAVone® signle-plasmid AAV production system. They integrates all the AAV production necessary components, including Ad helper genes (E2A, E4orf6 and VA RNA), AAV helper genes (Rep and Cap), and AAV genome. To produce AAVs, users only need select the pAAVone plasmid carrying the corresponding capsid, clone your transgenes into it and transfect them into host cells. Pre-made pAAVone plasmids are available in the following serotypes: AAV1, AAV2, AAV2-Retro, AAV3B, AAV4, AAV6, AAV7, AAV8, AAV9, AAV11, AAV12, AAV13, AAV-DJ, AAVrh.10, AAVrh.74, and AAV-PHP.eB.