29 February 2012 Last updated at 10:39 ET

Treatment which aims to correct a rare inherited genetic defect has helped a patient at risk from serious infection, a leading hospital is reporting.

The use of gene therapy against chronic granulomatous disorder (CGD) is a third success for Great Ormond Street Hospital in London.

Patients with CGD cannot make cells to fight bacterial and fungal infection.

Scientists used a virus to deliver a functioning version of the faulty gene which causes the disease.

Clinical trials at Great Ormond Street and its linked research centre, the Institute for Child Health at University College London, have focused on rare immune conditions caused by a single gene defect.

Early trials in 16 patients with X-linked severe combined immunodeficiency syndrome (x-SCID) and Adenosine Deaminase Deficiency causing Severe Combined Immune Deficiency (ada-SCID), who previously were so vulnerable to infection they needed to live in sterile conditions, have worked well, allowing most of them to start enjoying normal lives.

The decision was taken to use a similar technique on a teenage CGD patient who had fallen prey to a serious fungal lung infection, and was not expected to survive more than a year.

One in 150,000 children has the gene defect which causes CGD, and there is only one way to cure it - with a bone marrow transplant.

In the case of the teenage patient, no matching donor was available.

See the rest here:
Therapy 'aiding immune disease'

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