pAAVone-AAV5-CMV-EGFP-2.8kb

pAAVone-AAV5-CMV-EGFP-2.8kb is a pAAVone plasmid used to produce AAV5 Reference (Full) and AAV5 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 AAV5), and a 2.8kb single-plasmid strand AAV-CMV-EGFP genome. AAV5-CMV-EGFP viruses can be simply and efficiently produced by transfection of this single-into host cells, such us HEK 293 and HEK 293T. AAV5 is a serotype of adeno-associated virus that was originally isolated from rhesus monkeys, with unique tissue tropism and characteristics that make it useful in specific gene therapy applications. AAV5 has demonstrated an ability to transduce various tissues, including the brain, lungs, and liver. Gene therapy based on AAV5 has been approved for human use, such as Roctavian (valoctocogene roxaparvovec), developed by BioMarin Pharmaceutical for the treatment of hemophilia A.

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Background

Our data revealed that AAV5 delivers low levels of RNA compared with DNA across multiple tissues in C57BL/6 mice following intravenous (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.