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xFrench gene therapy success

22 April 2002
By BioNews
Appeared in BioNews 154

A report in last week's New England Journal of Medicine shows that children treated by gene therapy at the Necker Hospital for Sick Children in Paris, France, are still living normal lives after more than two years.

Four boys were successfully treated in the French study, all having the X-linked genetic condition severe combined immune deficiency (X-SCID). Boys affected by the life-threatening disorder have to live in sterile environments or 'bubbles' to avoid contracting any infections. X-SCID is the same condition that Rhys Evans, the toddler who was successfully treated with gene therapy at London's Great Ormond Street Hospital recently, suffered from.

The Necker study was originally reported in May 2000. To carry out the gene therapy treatment, the French researchers harvested bone marrow from the patients. They then isolated blood stem cells from the bone marrow, which they infected with a virus carrying a replacement gene. After three days of repeated gene transfers, the cells were transplanted back into the patients. The immune systems of two of the boys, then aged eight and eleven months, appeared to begin to function normally and they left their sterile environment to go home. Two other boys on the gene therapy program were later also allowed home.

According to the new report, the condition of the four boys has not deteriorated, leading some people to believe that these are the first humans to be cured by gene therapy. However, Marina Cavazzana-Calvo, from the Necker Hospital, remains cautious about the use of the word 'cured', saying 'all we can say without problem is that the effect is reproducible in other children, and lasts long-term'.

SOURCES & REFERENCES
French research offers promise of gene therapy cure
The Washington Post |  18 April 2002
Gene therapy restores more babies' immune systems
Yahoo Daily News |  17 April 2002
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