Gene lab probes cures for children

The lab hopes to unravel gene cures for children

A brand-new laboratory at one of the country's leading research hospitals is hoping to produce therapies for the most severe genetic diseases.

Researchers at Great Ormond Street Hospital in London are hoping to turn their work on possible gene cures into real-life treatments for sick children.

Some of the first clinical trials planned by the hospital are aimed at defects of the immune system.

One example is X-SCID - commonly referred to as "baby in the bubble syndrome" - in which children must live in a completely sterile environment because they have no immune system of their own.

The only current treatment is bone marrow transplantation, and many patients do not live beyond their first birthday.

However, the illness is caused by a single defective gene, which makes it a suitable target for gene therapy.

Dr Adrian Thrasher, a researcher into the genetics of immune deficiencies, said: "The real advances in genetic understanding may appear somewhat abstract to most people.

"Gene therapy is about turning understanding into real cures for real children. We hope that these two proposed trials are only the beginning of gene therapy at Great Ormond Street and the Institute of Child Health.

First of its type

Another disease which the researchers are aiming to treat with gene therapy is chronic granulomatous disease (CGD).

Children with the illness are vulnerable to infection because their immune cells do not work properly.

The Great Ormond Street trial will be the first of its type in the world, taking a small sample of bone marrow, then destroying the rest with chemotherapy before genetically modifying the sample and returning it.

The lab has been funded by the Jeans for Genes campaign.

The campaign has raised more than £9.5m since it started six years ago.

This has not only paid for research across the UK, but also the foundation of support groups for parents of children with genetic illnesses.