Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

by George, L.A., Sullivan, S.K., Giermasz, A., Rasko, J.E.J., Samelson-Jones, B.J., Ducore, J., Cuker, A., Sullivan, L.M., Majumdar, S., Teitel, J., McGuinn, C.E., Ragni, M.V., Luk, A.Y., Hui, D., Wright, J.F., Chen, Y., Liu, Y., Wachtel, K., Winters, A., Tiefenbacher, S., Arruda, V.R., van der Loo, J.C.M., Zelenaia, O., Takefman, D., Carr, M.E., Couto, L.B., Anguela, X.M., High, K.A.
Published in N Engl J Med (2017)

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

by Hacein-Bey-Abina, S., Pai, S.-Y., Gaspar, H.B., Armant, M., Berry, C.C., Blanche, S., Bleesing, J., Blondeau, J., de Boer, H., Buckland, K.F., Caccavelli, L., Cros, G., De Oliveira, S., Fernández, K.S., Guo, D., Harris, C.E., Hopkins, G., Lehmann, L.E., Lim, A., London, W.B., van der Loo, J.C.M., Malani, N., Male, F., Malik, P., Marinovic, M.A., McNicol, A.-M., Moshous, D., Neven, B., Oleastro, M., Picard, C., Ritz, J., Rivat, C., Schambach, A., Shaw, K.L., Sherman, E.A., Silberstein, L.E., Six, E., Touzot, F., Tsytsykova, A., Xu-Bayford, J., Baum, C., Bushman, F.D., Fischer, A., Kohn, D.B., Filipovich, A.H., Notarangelo, L.D., Cavazzana, M., Williams, D.A., Thrasher, A.J.
Published in N Engl J Med (2014)