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Gene Therapy

Gene Therapy Responsible for Leukemia in Bubble Boys

20 years, 4 months ago

8432  0
Posted on Dec 07, 2003, 2 a.m. By Bill Freeman

Researchers have confirmed that gene therapy which saved the lives of two boys suffering from so-called "bubble boy syndrome" was responsible for the children developing leukemia. Dr Alain Fischer of Necker Hospital in Paris and colleagues said that the experimental gene therapy triggered a gene that caused the boys' bone marrow to produce excessive amounts of immune system cells called T-cells.

Researchers have confirmed that gene therapy which saved the lives of two boys suffering from so-called "bubble boy syndrome" was responsible for the children developing leukemia. Dr Alain Fischer of Necker Hospital in Paris and colleagues said that the experimental gene therapy triggered a gene that caused the boys' bone marrow to produce excessive amounts of immune system cells called T-cells. The two boys were among a group of ten treated for a rare genetic disease called x-linked severe combined immunodeficiency or SCID-X1, which is caused by a defective gene. Fischer and colleagues used a virus to deliver a correct copy of the gene to the children. The treatment was seemingly successful and the children were cured of SCID, however by the time the children were approaching their third birthdays' doctors spotted that two of the children appeared to be developing symptoms of leukemia. Both boys have now been successfully treated with chemotherapy.

SOURCE/REFERENCE: Reported by www.reutershealth.com on the 16th October 2003.

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