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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...

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Detalles Bibliográficos
Autores principales:	Sivalingam, Jaichandran, Kenanov, Dimitar, Han, Hao, Nirmal, Ajit Johnson, Ng, Wai Har, Lee, Sze Sing, Masilamani, Jeyakumar, Phan, Toan Thang, Maurer-Stroh, Sebastian, Kon, Oi Lian
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2016
Materias:	Original Article
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

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