Phenotypic Correction of Hemophilia A in Sheep by Postnatal Intraperitoneal Transplantation of FVIII-Expressing MSC

We recently re-established a line of sheep that accurately mimics the clinical symptoms and genetics of severe hemophilia A (HA). Herein, we tested a novel, non-ablative transplant therapy in 2 pediatric HA animals. Paternal mesenchymal stem cells (MSC) were transduced with a porcine FVIII-encoding...

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Detalhes bibliográficos
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: 2011
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3220800/
https://ncbi.nlm.nih.gov/pubmed/21906573
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.exphem.2011.09.001
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