To date, more than 100 gene therapy clinical trials have been conducted, tackling a variety of diseases such as lipoprotein lipase (LPL) deficiency, cystic fibrosis, and hemophilic disorders (Carter 2005; Bryant et al. 2013; Gene therapy clinical trials website: http://www.abedia.com/wiley/). This evidence concerns the gene LPL and hyperinsulinemic hypoglycemia, familial, 4.