Complete correction of murine Artemis immunodeficiency by lentiviral vector-mediated gene transfer.

@article{Mostoslavsky2006CompleteCO,
  title={Complete correction of murine Artemis immunodeficiency by lentiviral vector-mediated gene transfer.},
  author={Gustavo Mostoslavsky and Attila J. Fabian and Sean Rooney and Frederick W Alt and Richard C. Mulligan},
  journal={Proceedings of the National Academy of Sciences of the United States of America},
  year={2006},
  volume={103 44},
  pages={16406-11}
}
Artemis gene mutations are responsible for the development of a severe combined immunodeficiency [radiation-sensitive (RS) SCID] characterized by a severe B and T cell deficiency and a normal natural killer cell population. To establish the feasibility of a gene therapy approach to the treatment of RS-SCID, we generated a series of lentiviral vectors expressing human Artemis from different promoters and used them to transduce highly purified hematopoietic stem cells (HSCs) from Artemis knockout… CONTINUE READING

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