pAAVone-AAV9-CMV-EGFP-2.8kb

pAAVone-AAV9-CMV-EGFP-2.8kb is a pAAVone plasmid used to produce AAV9 Reference (Full) and AAV9 Reference (Byproduct-Empty) using AAVnerGene’s 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 2.8kb 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.

• Full Plasmid Name: pAAVone-AAV9-CMV-SV40I-EGFP-WPRE-hGHpolyA-2.8kb
• AAV Capsid: AAV9
• Final AAV Genome: AAV-CMV-SV40I-EGFP-WPRE-hGHpolyA-2.8kb, 2843 bp
• Corresponding AAV vectors: AAV9 Reference (Full) and AAV9 Reference (Byproduct-Empty)
• AAV Genome Type: ssAAV
• ITRs:  L-ITR (119 bp) + R-ITR (130 bp)
• Promoter: CMV
• Transgene: EGFP
• Plasmid Size: 15,796 bp
• Bacterial Resistance(s) : Kana
• Plasmid Backbone: pAAVone
• Storage:  -20°C for long term (> 1 year)
• Shipping: Dry ice.
• Notice to Purchaser:  MTA is required. All mini-pHelper-1.0 based products, including mini-pHelper-1.0 itself, pAAVdual and pAAVone plasmids,  are sold under license of US 20240229067. Rights to use these products are limited to non-commercial research only. No other rights are conveyed. Inquiry into the availability of a license to broader rights or the use of these products for commercial purposes should be directed to AAVnerGene Inc, 9620 Medical Center Dr, Suite 100, Rockville, MD, 20850. Purchase of these products does not grant rights to : (1) offer the vectors or any derivatives thereof for resale; or (2) to distribute or transfer the vector or any derivative thereof to third parties.
• Cloning Services: AAVnerGene provides cloning services based on this plasmid backbone with $300/clone.
• AAV Package Services: AAVnerGene provides affordable AAV production services at various scales(from 1e12 to 1e16 vg), in both suspension and adherent cultured cells (HEK 293T or HEK 293), with flexible purification methods (Iodixanol, CsCl, or AAV-X resin).
• AAV Analytical Services: We also offer advanced AAV characterization services,  including AAV purity, vector genome identity, ITR integrity, DNA impurities, full/empty ratios, and a variety of comprehensive analyses based on custom requests.