The world’s first gene therapy – level 3
01-12-2025 07:00
Three-year-old Oliver Chu has become the first person in the world with Hunter syndrome, a rare inherited condition causing progressive damage to the body and brain, to receive a ground-breaking gene therapy developed in Manchester, UK.
The disease is devastating, often described as a form of childhood dementia, with patients in severe cases typically dying before the age of 20. Hunter syndrome is caused by a faulty gene that prevents the production of a crucial enzyme needed for healthy cells. To combat this, medical staff genetically altered Oliver’s stem cells by inserting a working copy of the gene via a harmless virus. This process instructs the cells to produce the missing enzyme, aiming to stop further physical and neurological damage. A year after receiving the one-off infusion, Oliver’s parents and doctors are astounded. He appears to be developing normally, with significant improvements in his speech, mobility, and cognitive abilities. He has been able to stop all other treatments, unlike his five-year-old brother, Skylar, who has the same condition but was too old for this trial. Researchers spent 15 years developing the therapy; however, they are not sure whether the treatment could be a permanent fix for the disease.
Difficult words: devastating (causing great damage, harm, or sadness), crucial (extremely important), stem cell (a special kind of cell in the body that has the power to develop into many different types of cells), one-off (happening only once; unique and not repeated).
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What improvements have been seen in Oliver’s speech, mobility, and cognitive abilities a year after his one-off infusion?
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