2006•Nature MedicineRequires access

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Catherine Scott Manno, Glenn F. Pierce, Valder R. Arruda, Bertil E. Glader, Margaret V. Ragni, John E.J. Rasko, Margareth Castro Ozelo, Keith W. Hoots, Philip M. Blatt, Barbara A. Konkle, Michael D. Dake, Robin D. Kaye, Mahmood Razavi, Albert Zajko, James L. Zehnder, Pradip K. Rustagi, Hiroyuki Nakai, Amy Chew, Debra G. B. Leonard, J Fraser Wright, Ruth Lessard, Jürg M. Sommer, Michael A. Tigges, Denise E. Sabatino, Alvin Y. Luk, Haiyan Jiang, Federico Mingozzi, Linda Couto, Hildegund C.J. Ertl, Katherine A. High, Mark A. Kay

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Key concepts: Factor IX, Medicine, Immune system, Asymptomatic, In vivo, Immunology, Genetic enhancement, Internal medicine