The emerging technique of genetic therapy is a promising therapeutic approach that has been or is currently attempted in a regimen of diseases: e.g., neurological disorders such as Huntington's disease [IONIS-HTTRx; ClinicalTrials.govidentifier: NCT02519036; (182)] and spinal muscular atrophy (SMA) (140), immunological diseases as severe combined immune deficiency due to adenosine-deaminase deficiency [ADA-SCID; (183)], cardiovascular diseases (184) and malignant diseases like acute lymphoblastic leukemia (185) or non-Hodgkin lymphoma (186). The gene discussed is ADA; the disease is acute lymphoblastic leukemia.