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As a journalist, you can create a free Muck Rack account to customize your profile, list your contact preferences, and upload a portfolio of your best work.Articles
rAAV8 encapsidated HMR-001/z enables high efficiency hepatic transduction and restores hemostasis in hemophilic mice
Abstract Hemophilia A (HA), an X-linked bleeding disorder caused by factor VIII (FVIII) deficiency, is primarily managed with exogenous therapeutic agents; however, this treatment approach remains burdensome and fails to provide durable hemostatic control. Adeno-associated viral (AAV) vectors enabling endogenous FVIII expression have emerged as promising alternatives to address these limitations, but existing vectors show limited transduction efficiency and declining activity over time.
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