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Replacing the haemophilia gene

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In August 2016, Lucina featured two new treatments likely to be highly effective in treating haemophilia (doi101136/archdischild-2016-31162). Now here’s another, even more radical, which promises to actually correct the genetic defect. The X-linked haemophilia gene, which spread to all the royal houses of Europe in the early 20th century by virtue of Queen Victoria’s fecundity, affected the heir to the Russian throne and was arguably responsible for the 1917 Russian revolution.

A successful gene therapy study involving patients with haemophilia …

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