Boy with rare condition amazes doctors after world-first gene therapy | BBC News
Three-year-old Oliver Chu has astounded doctors with his progress after becoming the first person in the world with his devastating disease to receive a ground-breaking gene therapy. "Every time we talk about it, I want to cry because it's just so amazing," says the boy’s mother, Jingru. He has a rare, inherited condition called Hunter syndrome - or MPSII - which causes progressive damage to the body and brain. In the most severe cases, patients with the disease usually die before the age of 20. The effects are sometimes described as a type of childhood dementia. Due to a faulty gene, before the treatment Oliver was unable to produce an enzyme crucial for keeping cells healthy. In a world first, medical staff in Manchester, UK, have tried to halt the disease by altering Oliver's cells using gene therapy. Now, a year after starting treatment - he appears to be developing normally.
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