Boy with rare disease amazes doctors after first-ever gene therapy
Kyiv • UNN
Three-year-old Oliver Chu, who has the rare Hunter syndrome, received revolutionary gene therapy in Manchester. A year after starting treatment, the boy is developing normally, which has amazed doctors.
A three-year-old boy amazed doctors with his progress, becoming the first person in the world with a severe rare disease - Hunter syndrome (MPSII) - to receive revolutionary gene therapy, reports the BBC, writes UNN.
Details
Oliver Chu has a rare hereditary disease called Hunter syndrome, which causes progressive damage to the body and brain.
In the most severe cases, patients with this disease usually die before the age of 20. Its effects are sometimes described as a type of childhood dementia.
Due to a defective gene, Oliver could not produce an enzyme necessary for healthy cells before treatment began.
For the first time in the world, medical professionals in Manchester tried to stop the disease by modifying Oliver's cells with gene therapy.
Professor Simon Jones, one of the research leaders, told the BBC: "I've waited 20 years to see a boy like Ollie doing so well, and it's so exciting."
At the heart of this remarkable story is Oliver - the first of five boys worldwide to receive the treatment - and the Chu family from California, who trusted the team of doctors at Manchester Royal Children's Hospital.
A year after starting treatment, Oliver appears to be developing normally.
"Every time we talk about it, I want to cry because it's so amazing," says his mother Jingru.