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Ultrasound-mediated gene delivery of factor VIII plasmids for hemophilia A gene therapy in mice

Gene therapy offers great promises for a cure of hemophilia A resulting from factor VIII (FVIII) gene deficiency. We have developed and optimized a non-viral ultrasound-mediated gene delivery (UMGD) strategy. UMGD of reporter plasmids targeting mice livers achieved high levels of transgene expressio...

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Detalles Bibliográficos
Autores principales:	Song, Shuxian, Lyle, Meghan J., Noble-Vranish, Misty L., Min-Tran, Dominic M., Harrang, James, Xiao, Weidong, Unger, Evan C., Miao, Carol H.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2022
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8803955/ https://www.ncbi.nlm.nih.gov/pubmed/35141050 http://dx.doi.org/10.1016/j.omtn.2022.01.006

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