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The World in Medicine
February 12, 2003

Gene Therapy Setback

JAMA. 2003;289(6):691. doi:10.1001/jama.289.6.691-a

For the second time, a child treated in a clinical trial with gene therapy to correct a genetic disorder has developed leukemia, the French Medical Products Safety Agency reported last month (http://agmed.sante.gouv.fr/htm/11/11000.htm). The trial, conducted by researchers at the Necker Hospital for Sick Children, in Paris, had been regarded as the first gene therapy effort to clearly demonstrate efficacy, with 9 of 11 boys apparently cured of X-linked severe combined immunodeficiency disease. The condition, often referred to as "bubble baby disease," involves severe defects in T-cell and B-cell immunity.