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Huntington’s disease is brutal in its simplicity. The disorder, which slowly bulldozes your ability to control your body, starts with just a single mutation, in the gene for huntingtin protein. That tweak tacks an unwelcome glob of glutamines—extra amino acids—onto the protein, turning it into a destroyer that attacks neurons.
Huntington’s simplicity is exciting, because theoretically, it means you could treat it with a single drug targeted at that errant protein. But in the 24 years since scientists discovered it the gene for huntingtin, the search for suitable drugs has come up empty. This century’s riches of genetic and chemical data seem like it should have sped up research, but so far, the drug pipeline is more faucet than fire hydrant.
Part - Problem - Drug - Design - Researchers
Part of the problem is simply that drug design is hard. But many researchers point to the systems of paywalls and patents that lock up data, slowing the flow of information. So a nonprofit called the Structural Genomics Consortium is countering with a strategy of extreme openness. They’re partnering with nine pharmaceutical companies and labs at six universities, including Oxford, the University of Toronto, and UNC Chapel Hill. They’re pledging to share everything with each other—drug wish lists, results in open access journals, and experimental samples—hoping to speed up the long, expensive drug design process for tough diseases like Huntington’s.
Rachel Harding, a postdoc at the University of Toronto arm of the collaboration, joined up to study the Huntington’s protein after she finished her PhD at Oxford. In a recent round of experiments, her lab grew insect cells in stacks of lab flasks fed with pink media. After slipping the cells a DNA vector that directed them to produce huntingtin, Rachel purified and stabilized the protein—and once it hangs out in a deep freezer for a while, she’ll map it with an...
(Excerpt) Read more at: WIRED
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