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An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation

9 Nov 2007

Abstract excerpt

Pathogenic activation of the LMO2 proto-oncogene by an oncoretroviral vector insertion in a clinical trial for X-linked severe combined immunodeficiency (X-SCID) has prompted safety concerns. We used an adeno-associated virus vector to achieve targeted insertion of a gamma-retroviral long terminal repeat (LTR) driving a GFP expression cassette with flanking loxP sites in a human T-cell line at the precise...

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Identifiers

PMID: 17991809
DOI: 10.1182/blood-2007-04-085506

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