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Delivery of full-length factor VIII using a piggyBac transposon vector to correct a mouse model of hemophilia A.

Viral vectors have been used for hemophilia A gene therapy. However, due to its large size, full-length Factor VIII (FVIII) cDNA has not been successfully delivered using conventional viral vectors. Moreover, viral vectors may pose safety risks, e.g., adverse immunological reactions or virus-mediate...

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Detalhes bibliográficos
Main Authors: Hideto Matsui, Naoko Fujimoto, Noriko Sasakawa, Yasuhide Ohinata, Midori Shima, Shinya Yamanaka, Mitsuhiko Sugimoto, Akitsu Hotta
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science (PLoS) 2014-01-01
Colecção:PLoS ONE
Acesso em linha:http://europepmc.org/articles/PMC4134236?pdf=render
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