Scientists use HIV viruses and DNA editing to cure deadly childhood 'bubble boy disease'

Mail Online | 9/21/1971 | Sam Blanchard Health Reporter For Mailonline
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Scientists have found a way to cure children with a rare 'bubble boy disease' which leaves them without an immune system.

A total of 10 children in the US have been treated with an experimental gene therapy which made them produce vital white blood cells for the first time.

Genes - Children - DNA - Body - HIV

The altered genes used to fix the children's defective DNA were delivered into the body using damaged HIV viruses.

Experts working on the project say the breakthrough is a first for children with this rare and life-threatening illness, and say only time will tell if it's a permanent cure.

Doctors - St - Jude - Children - Research

Doctors at St Jude's Children Research Hospital in Memphis, Tennessee, say they have 'cured' the infants who can now live normal lives.

Each of the patients has X-linked severe combined immunodeficiency (SCID-X1), a condition present from birth which stops a child's immune system working.

Illness - Boy - Disease - Patients - Isolation

The illness is also known as 'bubble boy disease' because patients have to be kept in isolation tanks to avoid common infections like colds, which could kill them.

'This therapy has cured the patients,' said Dr Ewelina Mamcarz, one of the study leaders. But she added only time will tell if the cure is permanent.

Patients - Toddlers - Vaccinations - Systems - Cells

'These patients are toddlers now,' she said, 'who are responding to vaccinations and have immune systems to make all immune cells they need for protection from infections as they explore the world and live normal lives.

'This is a first for patients with SCID-X1.'

Treatment - Children - Disease - Body - Infections

Without treatment, children with bubble boy disease usually die when they're one or two because their body can't fight off even the simplest of infections.

The condition affects around one in 200,000 babies, mostly boys, and was brought into the public eye by a boy called David Vetter, who was born in Texas in 1971.

David - Bone - Marrow - Transplant

David could only have been cured by a bone marrow transplant but he died shortly after...
(Excerpt) Read more at: Mail Online
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