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Stunning gene therapy breakthroughs are a riposte to our truth-tarnished times

There has been a surprising outbreak of the use of the c-word among medical researchers over the past few days. Normally cautious in their language, they have nevertheless been wielding the term “cure” when discussing the long-term potential of two separate treatments for inherited ailments that were announced last week. Such enthusiasm is striking.

In one case, scientists based at St Bartholomew’s, London – who have been working on the inherited bleeding disorder haemophilia A – outlined how they had used a virus to carry the gene for the blood-clotting chemical, factor VIII (which patients lack) to their livers. Production of the missing chemical was restored and their bleeding halted. The development, according to the World Federation of Hemophilia, now points “the way to a cure” for the condition, which affects around 400,000 people worldwide.

In the other case, scientists led by Professor Sarah Tabrizi, of University College London, revealed they had found a way to suppress the build-up of harmful proteins in patients suffering from the incurable degenerative condition, Huntington’s disease. Injections of the drug Ionis-HTTRx destroyed genetic messengers that directed the manufacture of these proteins. Dementia experts hailed the news as a “tremendous step forward” because it could be used not only to target proteins involved in Huntington’s but in other neural conditions, such as Alzheimer’s disease.

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