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Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo

1 Aug 2007

Abstract excerpt

We treated 10 children with X-linked SCID (SCID-X1) using gammaretrovirus-mediated gene transfer. Those with sufficient follow-up were found to have recovered substantial immunity in the absence of any serious adverse events up to 5 years after treatment. To determine the influence of vector integration on lymphoid reconstitution, we compared retroviral integration sites (RISs) from peripheral blood CD3(+) T...

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PMID: 17671654
DOI: 10.1172/jci31661

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