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US doctors rewrite DNA of infant with severe genetic disorder in medical first | Gene-editing breakthrough has potential to treat array of devastating genetic diseases soon after birth, scientists say
Gene-editing breakthrough has potential to treat array of devastating genetic diseases soon after birth, scientists say
Specialists at the Children’s Hospital of Philadelphia and the University of Pennsylvania started work as soon as the boy was diagnosed and completed the complex design, manufacture and safety testing of the personalised therapy within six months. Writing in the New England Journal of Medicine, the doctors described the painstaking process of identifying the specific mutations behind KJ’s disorder, designing a gene-editing therapy to correct them, and testing the treatment and fatty nanoparticles needed to carry it into the liver. Dr Miguel Ángel Moreno-Mateos, a geneticist at Pablo de Olavide University in Seville, said: “Although this has been a very specific approach, partly motivated by the devastating nature of the disease, it represents a milestone that demonstrates these therapies are now a reality.
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