Treatment of canine hemophilia A via intraosseous delivery of a platelet-specific factor VIII-lentiviral vector.

ABSTRACT: Hemophilia A (HemA) is a genetic disease resulting from a factor VIII (FVIII) deficiency. Traditional protein infusions to treat HemA are costly and inconvenient and require repeated dosing. We demonstrated previously that intraosseous (IO) gene therapy via delivery of lentiviral vectors (LVs) into bone marrow targeting FVIII expression in platelets successfully treated HemA mice. To facilitate the translation of this novel strategy to clinical application, we investigated the treatment of HemA dogs using IO gene therapy. The vesicular stomatitis virus-pseudotyped G-cF8-LV incorporating a platelet-specific promoter, Gp1bα, and canine F8 gene was injected into the tibia or iliac bones of 4 HemA dogs. All dogs recovered well from the procedure and had blood chemistry values within nor

SUBMITTER: Rementer CW 

PROVIDER: S-EPMC12835592 | biostudies-literature | 2026 Feb

REPOSITORIES: biostudies-literature