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

• Main Authors: Shuxian Song (Author), Meghan J. Lyle (Author), Misty L. Noble-Vranish (Author), Dominic M. Min-Tran (Author), James Harrang (Author), Weidong Xiao (Author), Evan C. Unger (Author), Carol H. Miao (Author)
• Format: Book
• Published: Elsevier, 2022-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...