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Multidimensional Genome-wide Analyses Show Accurate FVIII Integration by ZFN in Primary Human Cells
Costly coagulation factor VIII (FVIII) replacement therapy is a barrier to optimal clinical management of hemophilia A. Therapy using FVIII-secreting autologous primary cells is potentially efficacious and more affordable. Zinc finger nucleases (ZFN) mediate transgene integration into the AAVS1 locu...
Autores principales: | , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4786920/ https://www.ncbi.nlm.nih.gov/pubmed/26689265 http://dx.doi.org/10.1038/mt.2015.223 |