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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Détails bibliographiques
Auteurs principaux: Matsui, Hideto, Fujimoto, Naoko, Sasakawa, Noriko, Ohinata, Yasuhide, Shima, Midori, Yamanaka, Shinya, Sugimoto, Mitsuhiko, Hotta, Akitsu
Format: Artigo
Langue:Inglês
Publié: Public Library of Science 2014
Sujets:
Research Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4134236/
https://ncbi.nlm.nih.gov/pubmed/25126862
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0104957
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