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Clinical Implications of Basic Research
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Volume 350:1679-1680 April 15, 2004 Number 16
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Good News for Gene Therapy
Anton Berns, Ph.D.

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Gene therapy has endured several setbacks in recent years. One setback involved infants born with X-linked severe combined immunodeficiency (SCID) resulting from a mutation of the {gamma}c gene who were successfully treated with an infusion of autologous hematopoietic stem cells transduced with a defective retroviral vector.1 The enthusiasm generated by the apparent cure of 9 of the 10 infants turned to alarm when, nearly three years after treatment, T-cell leukemia developed in 2 of the boys.2 A recent study by Davé and colleagues,3 however, suggests that this setback may have been due to a coincidence and that it is possible . . . [Full Text of this Article]


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From the Division of Molecular Genetics, the Netherlands Cancer Institute, Amsterdam.


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