T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates

Abstract

Adenosine deaminase-deficient severe combined immunodeficiency was the first disease investigated for gene therapy because of a postulated production or survival advantage for genecorrected T lymphocytes, which may overcome inefficient gene transfer. Four years after three newborns with this disease were given infusions of transduced autologous umbilical… (More)
DOI: 10.1038/nm0798-775

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@article{Kohn1998TLW, title={T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates}, author={Donald B Kohn and Michal S. Hershfield and Denise A. Carbonaro and Ann O. Shigeoka and Judith S. Brooks and Elzbieta Monika Smogorzewska and Lora W. Barsky and Raymond Ming En Chan and Felix Burotto and Geralyn Annett and Jan A Nolta and Gay M. Crooks and Neena Kapoor and Melissa Eldetr and Diane W. Wara and Thomas Bowen and E. Birk Madsen and Floyd F. Synder and John Bastian and Linda Mesler Muul and R. Michael Blaese and Kenneth I. Weinberg and Robertson Parkman}, journal={Nature Medicine}, year={1998}, volume={4}, pages={775-780} }