Improving Gene Therapy Thirtyfold

When watching progress in medical science, you have to keep your eye on less flashy improvements in infrastructure and methodology. It is progress at that level that enables the later big, bright advances that capture all the attention. Here's an example of the type from ScienceDaily: "geneticists say they have developed a new version of the adeno-associated virus used in gene therapy that works about 30 times more efficiently in mice than vectors scientists currently rely on ... Based on our studies and those of others, it's become clear that the reason you need so much is because about half the [adeno-associated virus (AAV)] particles get stuck in the cytoplasm. It doesn't get to the nucleus very efficiently. The reason for that is obvious. AAV is seen by the body as an invading protein and it tries to block it ... We didn't change anything except the amino acid that does not allow phosphorylation to occur ... We were very surprised. It's amazing to think that changing one amino acid could produce these results." Gene therapy is a very important tool, and order of magnitude improvements in cost and efficiency here will ripple out through the cutting edge of medical research - including many areas important to the longevity medicine of tomorrow.



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