Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients.

J Howe, Steven; R Mansour, Marc; Schwarzwaelder, Kerstin; Bartholomae, Cynthia; Hubank, Michael; Kempski, Helena; H Brugman, Martijn; Pike-Overzet, Karin; J Chatters, Stephen; de Ridder, Dick; C Gilmour, Kimberly; Adams, Stuart; I Thornhill, Susannah; L Parsley, Kathryn; J T Staal, Frank; E Gale, Rosemary; C Linch, David; Bayford, Jinhua; Brown, Lucie; Quaye, Michelle; Kinnon, Christine; Ancliff, Philip; K Webb, David; Schmidt, Manfred; von Kalle, Christof; Bobby Gaspar, H; J Thrasher, Adrian

doi:10.1172/jci35798

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Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients.

Shared by NobleBlocks on Sep 1, 2008 • 12:00 AM UTC

Authors:

Steven J Howe

Marc R Mansour

Kerstin Schwarzwaelder

Abstract

X-linked SCID (SCID-X1) is amenable to correction by gene therapy using conventional gammaretroviral vectors. Here, we describe the occurrence of clonal T cell acute lymphoblastic leukemia (T-ALL) promoted by insertional mutagenesis in a completed gene therapy trial of 10 SCID-X1 patients. Integrati...

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