authors Konkle, B Coffin, D Naccache, M Youttananukorn, T Clark, C George, L Iorio, Alfonso Miesbach, W Noone, D Peyvandi, F Pipe, S Recht, M Skinner, M Valentino, L Mahlangu, J Pierce, G
keywords 32 Biomedical and Clinical Sciences 3206 Medical Biotechnology Biotechnology Gene Therapy Genetics Hematology Orphan Drug Rare Diseases