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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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Main Authors: | , , , , , , , |
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Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
Public Library of Science (PLoS)
2014-01-01
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Colecção: | PLoS ONE |
Acesso em linha: | http://europepmc.org/articles/PMC4134236?pdf=render |
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