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Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model

Robert A. French, Benjamin J. Samelson-Jones, Glenn P. Niemeyer, Clinton D. Lothrop, Jr, Elizabeth P. Merricks, Timothy C. Nichols, Valder R. Arruda
Journal: Blood Advances
Blood Adv (2018) 2 (5): 505–508.
https://doi.org/10.1182/bloodadvances.2017015313
Published: 2018
Includes: Supplemental data