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Adeno-Associated virus (AAV) is a small replication-defective, non-enveloped virus with single strand DNA genome. AAV virus can infect many non-dividing and dividing cell types for long-term gene expression without insertional mutagenesis, which makes AAV to be employed in gene therapy as a comparative safe and effective vector. Epidemiological studies have showed that 80% of healthy human are seropositive for AAV, which suggests AAV might have evolved an ideal relationship with its human host.
Recently, several gene therapy products, Kymriah to treat ALL, GALGT2 gene therapy to treat DMD, Luxturna to treat retinal dystrophy, Zolgensma to treat of SMA (Spinal Muscular Atropy) were approved by FDA in Unite State. With the advances of AAV vector-mediated gene therapies in researches, pre-clinic, clinic, it is reasonable that more gene therapy products would be approval and commercialized.
Beyond Biotech employs helper free system to package rAAV virus by co-transfection of three plasmids (rAAV-transgene, pAAV-Helper, pAAV-Rep/Cap) into HEK293 cells (Fig at right). Helper free system produces rAAV containing your gene of interest with a Helper plasmid (express Ad E2A, E4, VA RNA) instead of a helper adenovirus, which make your rAAV more yield, safe and effective.
We are dedicated to providing world-widely service of packaging the high quality AAV products and services to our scientific community with Adenovirus-free Hek293 system, please do not hesitate to contact and allow us the opportunity to work together with your successful research projects.
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