
Anna R. Sternberg
Featured in:
biorxiv.org
Articles
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May 21, 2024 |
biorxiv.org | Robert Davidson |Xueyuan Liu |Anna R. Sternberg |Cristina Martos-Rus
AbstractDurable factor VIII (FVIII) expression that normalizes hemostasis is an unrealized goal of hemophilia A adeno-associated virus (AAV)-mediated gene therapy. Trials with initial normal FVIII activity observed unexplained year-over-year declines in expression while others reported low-level, stable FVIII expression inadequate to restore normal hemostasis.
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