Eligibility for the Ex-Vivo Gene Therapy Trial

  • Both patients P1 aged 11 months and P2 aged 8 months did not have a bone marrow matched sibling.
  • Although P1 expressed the gamma-c receptor on the membrane, the receptor lacked the tail needed for communication. Thus, the gamma-c receptor, although present was functionless.
  • P2 did not express the protein at all.


  1. Both patients were placed in a sterile isolation ward. They received oral antibiotics and intravenous immunoglobulins every 3 weeks for 3 months.
  2. CD34+ cells from their bone marrow were harvested.
  3. After CD34+ cell separation, the cells were infected with the MFG gamma-c vector daily for 3 days.
  4. These cells were then given back to the patients.