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Editors Selection IGR 8-3
Basic research: Gene therapy
Comment by Curtis Brandt on:
14268 Mechanisms of AAV transduction in glaucoma-associated human trabecular meshwork cells, Borras T; Xue W; Choi VW et al., Journal of Gene Medicine, 2006; 8: 589-602
Viral mediated gene delivery to the eye has great potential for treating a number of diseases. Vectors based on Adeno-associated virus (AAV) have been shown to mediate long term therapeutic effects in animal models of retinal degenerative diseases and clinical trials are pending. Previous work has shown that AAV vectors based on serotype 2; do not transduce trabecular meshwork (TM) cells and gene delivery for glaucoma has focused on other delivery systems. The reason for the failure of AAV-2 mediated transduction of TM cells was thought to be due to a lack of receptors for the virus on the cells. Borras et al. (844) have examined this issue in detail. These carefully conducted studies demonstrate that AAV-2 vectors attach to and enter TM cells, thus receptor specificity is not the problem. AAV packages a single strand of DNA that must be converted to a double strand by host cell enzymes before the transgene can be expressed and this is where the transduction defect lies. This information is significant in thatit explains why TM cells are not transduced. A second significant finding of the work is that a self-annealing vector that forms a double stranded genome after entry corrects the defect and results in transduction of TM cells. The paper also presents microarray data on changes in host cell gene expression following transduction.
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