The ADA human gene therapy clinical protocol.

  title={The ADA human gene therapy clinical protocol.},
  journal={Human gene therapy},
  volume={1 3},
  • Published 1990 in Human gene therapy
Severe combined immunodeficiency (SCID) due to deficiency of the purine metabolic enzyme adenosine deaminase (ADA) is a fatal childhood immunodeficiency disease. Immune reconstitution by transplantation with HLA-identical bone marrow is the treatment of choice. For patients not candidates for bone marrow transplantation, we propose to attempt immune… CONTINUE READING