Adeno-associated virus (AAV)–mediated gene therapy is under investigation as a thera- peutic option for persons with hemophilia A. Efficacy and safety data include 3 years of follow-up after a single administration of AAV5-hFVIII-SQ
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M. Laffan | K. Pasi | S. Rangarajan | G. Pierce | C. Russell | W. Wong | B. Madan | E. Symington | N. Mitchell