Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with CD34+ selected autologous peripheral blood cells transduced with a human ADA gene. Amendment to clinical research project, Project 90-C-195, January 10, 1992.

@article{Blaese1993TreatmentOS,
  title={Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with CD34+ selected autologous peripheral blood cells transduced with a human ADA gene. Amendment to clinical research project, Project 90-C-195, January 10, 1992.},
  author={R. Michael Blaese and Kenneth W. Culver and L Chang and W. French Anderson and C V Mullen and Arthur Nienhuis and Charles Carter and Cynthia Dunbar and Susan F. Leitman and Mette Berger},
  journal={Human gene therapy},
  year={1993},
  volume={4 4},
  pages={521-7}
}
Significant increases in lymphocyte adenosine deaminase activity, T cell numbers and immune function have been achieved in the two children with SCID thus far treated with autologous T cells genetically-corrected by retroviral-mediated insertion of a normal ADA gene. Although the data obtained to date demonstrate that the use of ADA gene corrected peripheral T cells appears to be an effective treatment for ADA(-)SCID, it is theoretically preferable to try to develop a treatment for these… CONTINUE READING
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