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

Catherine S. Manno, Valder R. Arruda, Glenn F. Pierce, Bertil Glader, Margaret Ragni, John Rasko, Margareth C. Ozelo, Keith Hoots, Philip Blatt, Barbara Konkle, Michael Dake, Robin Kaye, Mahmood Razavi, Albert Zajko, James Zehnder, Hiroyuki Nakai, Amy Chew, Debra Leonard, J. Fraser Wright, Ruth R. LessardJürg M. Sommer, Michael Tigges, Denise Sabatino, Alvin Luk, Haiyan Jiang, Federico Mingozzi, Linda Couto, Hildegund C. Ertl, Katherine A. High, Mark A. Kay

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Biochemistry, Genetics and Molecular Biology

Neuroscience

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Immunology and Microbiology

Pharmacology, Toxicology and Pharmaceutical Science