Category Archives: Gene therapy

In this study, we sought to examine whether this attribute could be exploited in cancer suicide gene therapy by using a lentiviral (LV) vector expressing herpes simplex virus thymidine kinase (HSV-TK/GCV) under the regulation of antioxidant response element (ARE), a cis-acting enhancer sequence that binds Nrf2. In human lung adenocarcinoma cells in which Nrf2 is constitutively overexpressed, ARE activity was found to be high under basal conditions. In this setting, ARE-HSV-TK was more effective than a vector in which HSV-TK expression was driven by a constitutively active promoter. In a mouse xenograft model of lung cancer, suicide gene therapy with LV-ARE-TK/GCV was effective compared with LV-PGK-TK/GCV in reducing tumor size. We conclude that ARE-regulated HSV-TK/GCV therapy offers a pro...

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MicroRNAs are short non-coding RNAs that can regulate gene expression in cancer development, which makes them valuable targets for therapeutic intervention. Here we report on an approach that can not only arrest the functions of mature miRNAs by binding to them but it can also induce the “mis-processing” of the target miRNA producing a non-functional truncated miRNA. This approach involves generating an expression construct that produces a RNA fragment with sixteen repeat sequences. The construct is named miR-Pirate or microRNA-interacting RNA—producing imperfect RNA and tangling endogenous miRNA. The transcript of the construct contained mismatches to the seed region, and thus it would not target the potential targets of the miRNA under study. The homology of the cons...

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Scientists and engineers dream about big advances that could change the world, and then they try to create them. On the following pages, Scientific American reveals 10 innovations that could be game changers: an artificial alternative to DNA, oil that cleans water, pacemakers powered by our blood, and more. These are not pie-in-the-sky notions but practical breakthroughs that have been proved or prototyped and are poised to scale up greatly. Each has the potential to make what may now seem impossible possible. -- The Editors

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Yoshiki Sasai is not just an ordinary tissue engineer who tries to coax stem cells to grow into fully formed bodily structures. It is true that Sasai has made his mark by taking on big projects like using stem cells to whip up a retina, cortical tissue and the cerebellum, involved with balance and movement. But his research has gone deeper by delving into the way stem cells organize themselves into complex structures under the influence of genes and the prenatal environment. Read a profile of Sasai here to accompany “ Grow Your Own Eye ,” Sasai’s own account of growing a retina in the November Scientific American .

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In the womb, a ball of identical cells gives rise to varied cell types that ultimately form highly ordered structures and then the full panoply of organs in the human body. The process advances according to an internal biological script that directs each fold and crease of tissue to assume exactly the proper shape and dimension.

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The Press Release from the Nobel Assembly at Karolinska Institute

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