Stable expression of factor VIII over 5 years following adeno-associated viral vector-mediated gene transfer in participants with severe haemophilia a using a novel human factor VIII variant

P. Chowdary,U. Reiss,E. G. Tuddenham,J. McIntosh,P. Batty, J. C. M. Calvert,A. Riddell, A. Pie, R. Peralta, U. Dissanayake, K. Kamel, B. R. Sreedhar, A. J. Evans, V. Radulescu, E. Chang,M. A. Laffan,G. Kang, Y. Olufadi, M. M. Meagher, J. Zhou,C. L. Morton,A. M. Davidoff,A. C. Nathwani

HAEMOPHILIA（2024）

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