ArticleBlood vessels, thrombosis & hemostasis2026
Treatment of canine hemophilia A via intraosseous delivery of a platelet-specific factor VIII-lentiviral vector.
Article in Blood vessels, thrombosis & hemostasis, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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14 authors.
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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
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