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HS-CODE
S12-
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Detailed Description
Adeno-associated virus (AAV) has become the gene delivery system of choice in many gene therapy protocols. AAV has a broad range of infectivity, including both dividing and nondividing cells, and appears to be nonpathogenic. Greater than 80% of adults are seropositive for antibodies to AAV, yet no disease has been attributed to infection. Wild-type AAV demonstrates a proclivity for site-specific integration in healthy host cells, a particularly attractive feature for applications in gene therapy. The AAV genome is small, easy to manipulate, and substitution of all viral-encoded genes with a transgene of choice has been possible. The cloning of such recombinant AAV(rAAV), and development of in vitro packaging systems, have opened the doors for the development of an efficient DNA carrier system, free from concerns of recombination with wild-type virus, toxicity, and immunologic host response.
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