Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX

Brian Bigger, E. K. Siapati, A. Mistry, S. N. Waddington, M. S. Nivsarkar, L. Jacobs, R. Perrett, M. V. Holder, C. Ridler, G. Kemball-Cook, R. R. Ali, S. J. Forbes, C. Coutelle, N. Wright, M. Alison, A. J. Thrasher, D. Bonnet, M. Themis

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