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Article

Phenotypic correction of hemophilia A in sheep by postnatal intraperitoneal transplantation of FVIII-expressing MSC.

Experimental hematology - 1 Dec 2011

Porada Christopher D, Sanada Chad, Kuo Chung-Jung, Colletti Evan, Mandeville Walter, Hasenau John, Zanjani Esmail D, Moot Robert, Doering Christopher, Spencer H Trent, Almeida-Porada Graça

Abstract excerpt

We recently re-established a line of sheep that accurately mimics the clinical symptoms and genetics of severe hemophilia A (HA). Here, we tested a novel, nonablative transplantation therapy in two pediatric HA animals. Paternal mesenchymal stem cells (MSC) were transduced with a porcine FVIII-encoding lentivector and transplanted via the intraperitoneal route without preconditioning. At the time of...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 21906573
DOI: 10.1016/j.exphem.2011.09.001
PMCID: PMC3220800

Genes

MSC

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