Team:Heidelberg/Project/Mouse Infection
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==Introduction== | ==Introduction== | ||
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+ | The importance of virus shuttles for human gene therapy canot be understated. The Adeno-associated virus (AAV) is a non-pathogenic virus that can infect human tissue but does not replicate without a helper virus (like Adenovirus or Herpex simplex virus). Therefore it is ideal for the delivery of transgenes in vivo. AAVs are small (4.8kb genome size), nonenveloped viruses that exist in various natural serotypes with different properties in tropism and transduction speed and efficiency. The most intensively studied AAV serotype is AAV2, which is used in several ongoing clinical trials aiming at muscle tissue or central nervous system diseases. <br> | ||
+ | AAV viruses are easily manipulated, as they do not require any viral genes in cis (that is, on the same construct) genes needed for packaging (cap) and other structures (rep) that usually make up the viral genome can be delivered in trans from a second vector that is co-transfected. The only requirement for a gene to be packaged into an AAV virus is that it has to be flanked by Inverted Terminal Repeats (ITR) that serve as a packaging signal. | ||
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+ | For delivery of our transgenes into mice, we chose AAV serotype 8 as a carrier. This serotype, which has been isolated from rhesus monkey, has been shown to infect rapidly and especially target heart and liver. Since we are using the liver-specific miRNA 122 for On- and Off-targeting, this would be our ideal vector. | ||
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===gene therapy=== | ===gene therapy=== | ||
====Adeno-associated viruses==== | ====Adeno-associated viruses==== |
Revision as of 12:35, 27 October 2010
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