Delivery of Full-Length Factor VIII Using a piggyBac Transposon Vector to Correct a Mouse Model of Hemophilia A

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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Bibliographic Details
Main Authors: Matsui, Hideto, Fujimoto, Naoko, Sasakawa, Noriko, Ohinata, Yasuhide, Shima, Midori, Yamanaka, Shinya, Sugimoto, Mitsuhiko, Hotta, Akitsu
Format: Online
Language:English
Published: Public Library of Science 2014
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4134236/

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https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4134236/

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