Tuesday, February 3, 2009

Gene therapy curesa a "bubble boy disase"

I used to think that Gene Therapy was getting closer to curing diseases--now it seems to be there, for at least one disease.

Researchers report that Gene therapy seems to have cured 8 of 10 children who had potentially fatal "bubble boy disease" according to a study that followed their progress for about four years after treatment.

The eight patients were no longer on medication for the rare disease, which cripples the body;s defenses against infection.

The successful treatment is reported in Thursday's issue (January 29th, 2009) of the New England Journal of Medicine and offers hope for treating other diseases with a gene therapy approach.

Bubble boy disease is formally called sever combined immunodeficiency, or SCID. this genetic disorder is diagnosed in about 40 to 100 babies each year in the United States.

The nickname comes from the experience of a Houston boy, David Vetter, who became famous for living behind plastic barriers to protect him from germs. He died in 1984 at age 12.

He was portrayed in a made for TV film my John Travolta.

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