Improvement of helper-dependent adenovirus vector not only increasing gene expression but also cell/tissue specificity

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The helper-dependent adenovirus vector (HD-AdV) is attractive tool for gene therapy, specially, genetic disease, because of its low-inflammation. However, it was reported that the purpose gene expression efficiency is lower than E1-substituted AdV. In this study, we determined the useful stuffer region and demonstrated an only 0.3kb DNA region near the rightend of the virus genome played an important role for efficient gene expression. And we showed that this region did not code any protein and enhancer.We also developed a construction method of "cell/tissue-specific HD-AdV", which contains a switch unit carrying Cre gene expressed by cell/tissue-specific promoter and a target unit bearing purpose gene expression unit regulated by Cre. We chose the TH promoter for the cell/tissue-specific promoter and GFP for purpose gene. Finally, we succeeded in the construction and detected the dopamine neuron specific expression by this vector.