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Article

Intravenous injection of a foamy virus vector to correct canine SCID-X1

19 Mar 2014

Abstract excerpt

Current approaches to hematopoietic stem cell (HSC) gene therapy involve the collection and ex vivo manipulation of HSCs, a process associated with loss of stem cell multipotency and engraftment potential. An alternative approach for correcting blood-related diseases is the direct intravenous administration of viral vectors, so-called in vivo gene therapy. In this study, we evaluated the safety and efficacy of in...

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PMID: 24642749
DOI: 10.1182/blood-2013-11-538926

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