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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Detalhes bibliográficos

Principais autores:	Shuxian Song, Meghan J. Lyle, Misty L. Noble-Vranish, Dominic M. Min-Tran, James Harrang, Weidong Xiao, Evan C. Unger, Carol H. Miao
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2022-03-01
coleção:	Molecular Therapy: Nucleic Acids
Assuntos:	ultrasound microbubble hemophilia gene delivery factor VIII ultrasound mediated gene delivery
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S2162253122000117
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