pAAVone-AAV9-CMV-EGFP-2.2kb

pAAVone-AAV9-CMV-EGFP-2.2kb is a pAAVone plasmid used to produce AAV9-CMV-EGFP 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 AAV9), and a 2.2kb single-strand AAV-CMV-EGFP genome. AAV9-CMV-EGFP viruses can be simply and efficiently produced by transfection of this single-plasmid into host cells, such us HEK 293 and HEK 293T. AAV9 is one of the most widely used adeno-associated virus serotypes, especially known for its ability to efficiently transduce a broad range of tissues, including the central nervous system (CNS), liver, heart, and muscle. AAV9 has garnered considerable attention in the field of gene therapy due to its unique ability to cross the blood-brain barrier (BBB) and efficiently transduce neurons following systemic administration. This distinctive feature makes it particularly well-suited for both peripheral and central nervous system (CNS) gene therapy applications. A prominent example of its use is in Zolgensma (Onasemnogene Abeparvovec), a groundbreaking treatment for spinal muscular atrophy (SMA). SMA is a severe genetic disorder characterized by the loss of motor neurons and progressive muscle weakness.

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Background

Our data showed that AAV9 delivers high levels of RNA and DNA across various 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.