Bibliografía: 1. Mavilio, F. (2015). Gene therapy for inherited diseases of the blood. Hematology, 2015(1), 631-636. 2. Mendell, J. R., Rodino-Klapac, L. R., Sahenk, Z., Roush, K., Bird, L., Lowes, L. P., ... & Kaspar, R. (2013). Eteplirsen for the treatment of Duchenne muscular dystrophy. Annals of Neurology, 74(5), 637-647. 3. Cavazzana-Calvo, M., Payen, E., Negre, O., Wang, G., Hehir, K., Fusil, F., ... & Leboulch, P. (2010). Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature, 467(7313), 318-322. 4. Baylis, F., & McLeod, M. (2017). First-in-human phase 1 CRISPR gene editing cancer trials: are we ready? Current Gene Therapy, 17(4), 309-319.