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Drug that rewrites genes offers hope to children

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A pioneering therapy that cured a Belgian girl of a deadly immune disorder has been cleared for use in Britain

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He has spent the whole of his young life close to death, but Henry Vinen and children like him, born with a rare and deadly immune deficiency disease, may soon have hope of a cure thanks to the first gene therapy available on the NHS.

Next month, the UK’s National Institute for Health and Care Excellence (Nice) will tell hospitals that patients with conditions like Henry’s can have their faulty DNA rewritten.

It follows the cure of Margaux Moreels, a four-year-old Belgian girl born with a similar deadly genetic defect, who seemed doomed to the shortest of lives. “Just weeks after Margaux was born she contracted a lung infection and had to be ambulanced to hospital. It was terrifying,” her mother, Marijike, recalls.

After her treatment with the gene- altering drug Strimvelis, however, she is living the life of a typical girl and is about to start school.

Margaux and Henry both have forms of severe combined immunodeficiency (SCID), a gene disorder that causes defective white blood cells. Such disorders are caused by single mutations in the DNA of one gene. Many people carry such genes, inherited from one parent, without knowing. This is because a normal gene, from the other parent, masks its failings.

https://www.thetimes.co.uk/edition/news/drug-that-rewrites-genes-offers-hope-to-children-ht78vkxcb

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