Angela Huttner.

Selidji T. Agnandji, M facts .D., Angela Huttner, M.D., Madeleine E. Zinser, M.D., Patricia Njuguna, M.Med., Christine Dahlke, Ph.D. Fernandes, M.D., Sabine Yerly, M.Sc.D., Verena Kraehling, Ph.D., Rahel Kasonta, Ph.D., Akim A. Adegnika, M.D., Ph.D., Marcus Altfeld, M.D., Ph.D., Floriane Auderset, Ph.D., Emmanuel B. Bache, B.N.S., P.G.Cert., Nadine Biedenkopf, Ph.D., Saskia Borregaard, Ph.D., Jessica S. Brosnahan, M.H.Sc., Rebekah Burrow, B.Sc., Christophe Combescure, Ph.D., Jules Desmeules, M.D., Markus Eickmann, Ph.D., Sarah K. Fehling, Ph.D., Axel Finckh, M.D., Ana Rita Goncalves, Ph.D., Martin P. Grobusch, M.D., Ph.D., Jay Hooper, Ph.D., Alen Jambrecina, M.D., Anita L. Kabwende, M.D.D., Ph.D., Domtila Kimani, B.Sc., Bertrand Lell, M.D.Sc., Ansgar W.

Previous gene-therapy research with mature lymphocytes30 or hematopoietic stem cells17,18,22 indicated that enzyme-replacement therapy inhibited the outgrowth of ADA-transduced cells. Our study helps the notion a toxic environment due to high levels of purine metabolites at the time of stem-cell engraftment is beneficial in supporting the differential growth of gene-corrected cells, in lymphoid lineages especially. Gene therapy restored normal immune function in five patients and resulted in significant improvement in lymphocyte counts and features in the additional five patients, resulting in security from infectious complications.