ADA and hyperinsulinemic hypoglycemia, familial, 4: For this, the adenosine deaminase deficiency (ADA)-/- neonatal mouse model was used in preclinical studies to assess efficiency and safety of ADA-carrying lentiviral vectors by ex vivo transduction and the autologous transplantation of modified HSPCs, efforts that ultimately led to the EU marketing authorization of Strimvelis in 2016 [128,129,130].