The treatment is personalized for each person; hematopoietic stem cell (HSCs) are extracted from the person and purified so that only CD34-expressing cells remain. Those cells are cultured with cytokines and growth factors and then transduced with a gammaretrovirus containing the human adenosine deaminase gene and then reinfused into the person. These cells take root in the person's bone marrow, replicating and creating cells that mature and create normally functioning adenosine deaminase protein, resolving the problem. As of April 2016, the transduced cells had a shelf life of about six hours. Prior to extraction, the person is treated with granulocyte colony-stimulating factor in order to increase the number of stem cells and improve the harvest; after that but prior to reinfusion, the person is treated with busulfan or melphalan to kill as many of the person's existing HSCs to increase the chances of the new cells' survival.
Strimvelis
Angor animi
Aneugen
Troppenkoller
Cloforex
Anodyne
Cervarix
Acria