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Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors
Current treatment of hemophilia A (HemA) patients with repeated infusions of factor VIII (FVIII; abbreviated as F8 in constructs) is costly, inconvenient, and incompletely effective. In addition, approximately 25 % of treated patients develop anti-factor VIII immune responses. Gene therapy that can...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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BioMed Central
2016
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5056462/ https://www.ncbi.nlm.nih.gov/pubmed/27766066 http://dx.doi.org/10.1186/s12959-016-0105-1 |