Treatment that is "beating" rare syndrome! Scientists insert IDS gene into the body of a 3-year-old with "Hunter"

2025-11-24 14:32:01 / MISTERE&KURIOZITETE ALFA PRESS

Treatment that is "beating" rare syndrome! Scientists insert IDS gene

A three-year-old boy with the rare Hunter syndrome has shown remarkable improvement after doctors in Manchester inserted a healthy version of the IDS gene that was missing from his stem cells due to the disease. The case is still under observation but is considered the most successful treatment for the syndrome to date.

A remarkable medical breakthrough has taken place in Manchester, where a three-year-old California boy, Oliver Chu, has become the first person in the world with the rare Hunter syndrome to be treated with a new gene therapy.

This inherited disease causes progressive damage to the body and brain and is usually fatal before the age of 20.

In December 2024, doctors at the Royal Manchester Children's Hospital took stem cells from Oliver's blood.

In the laboratory in London, scientists introduced into them a healthy version of the IDS gene, which is missing in patients with Hunter syndrome.

This new gene was introduced using a modified virus, incapable of causing disease.

The "repaired" cells were returned to Oliver with two infusions in February 2025.

These cells could continuously produce the vital enzyme that he previously did not produce and prevent the progression of the disease, even in the brain, which existing treatments could not do.

Just three months after treatment, Oliver showed significant improvement in speech and movement. He has become more active, more attentive, and more sociable.

Subsequently, tests confirmed that he already produces hundreds of times more enzyme than a healthy person.

For the first time since his diagnosis, Oliver no longer needs the weekly infusions, which cost around 300,000 euros a year and did not protect his brain.

Doctors say Oliver is developing like a normal child, but emphasize that the real test is follow-up over the next two years.

So far, the signs are better than could have been imagined.

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