A Serious Adverse Event after Successful Gene Therapy for X-Linked Severe Combined Immunodeficiency

doi:10.1056/NEJM200301163480314

Volume 348:255-256		January 16, 2003		Number 3

A Serious Adverse Event after Successful Gene Therapy for X-Linked Severe Combined Immunodeficiency

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by Noguchi, P.

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To the Editor: We recently reported (April 18 issue)¹ the sustainedcorrection of X-linked severe combined immunodeficiency diseaseby ex vivo, retrovirally mediated transfer of the ${gamma}$ c gene intoCD34+ cells in four of five patients with the disease. Theseresults have since been confirmed in four additional patientswith typical X-linked severe combined immunodeficiency. Of thefirst four successfully treated patients, three continue todo well up to 3.6 years after gene therapy, whereas a seriousadverse event occurred in the fourth patient. At a routine checkup30 months after gene therapy, lymphocytosis consisting of amonoclonal population of . . . [Full Text of this Article]

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