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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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Detalles Bibliográficos
Autor principal: Miao, Carol H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2016
Materias:
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