Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients.

@article{Bordignon1995GeneTI,
  title={Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients.},
  author={Claudio Bordignon and Luigi D Notarangelo and N Nobili and Giuliana Ferrari and Giulia Casorati and Paola Panina and Evelina Mazzolari and Dario Maggioni and Claudia Rossi and Paolo Servida and Alberto G. Ugazio and Fulvio Mavilio},
  journal={Science},
  year={1995},
  volume={270 5235},
  pages={470-5}
}
Adenosine deaminase (ADA) deficiency results in severe combined immunodeficiency, the first genetic disorder treated by gene therapy. Two different retroviral vectors were used to transfer ex vivo the human ADA minigene into bone marrow cells and peripheral blood lymphocytes from two patients undergoing exogenous enzyme replacement therapy. After 2 years of treatment, long-term survival of T and B lymphocytes, marrow cells, and granulocytes expressing the transferred ADA gene was demonstrated… CONTINUE READING
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