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