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Cracking gene therapy tells the heroic human story behind the licensing of the worlds first major pharmaceutical gene therapy strimvelis the moving account of margaux a baby girl on todays groundbreaking trial is interwoven with a historical journey through the triumphs and tribulations of the field. The worlds first course of gene therapy began last week doctors in bethesda maryland infused white blood cells back into a four year old girl after genetically altering them to correct a . The first patient to be treated with gene therapy was a four year old girl treated at the nih clinical center in 1990 she had a congenital disease called adenosine deaminase ada deficiency . On september 14 1990 the first approved gene therapy procedure was performed on four year old patient see video 1 born with a rare genetic disease called she lacked a healthy immune system and was vulnerable to every passing germ or infectionchildren with this illness usually develop overwhelming infections and rarely survive to adulthood a common childhood illness is life threatening