Question

Give various steps in the gene therapy to cure ADA deficiencу.

Answer 1

1. Bone marrow stem cells that produce lymphocytes are collected from the patient.
2. A functional ADA gene is inserted into a harmless, engineered retroviral vector.
3. The vector is used to introduce the gene into the patient’s stem cells.
4. The genetically modified stem cells are returned to the patient’s bone marrow.
5. The cells produce ADA and can continue to multiply; introducing the gene into long-lasting stem cells can provide a more sustained benefit.