"Learn and Know" in the era of gene addition/gene editing based approaches for sickle cell disease: The EHA-EBMT consensus paper

“Then you better start swinnin’ or you’ll sink like a stone. For the times they are a-changin” RZ (Bob Dylan, Nobel prize in literature). But: Adso: “Then, why do you want to know?” William of Baskervile: “Because learning does not consist only of knowing what we must or we can do, but also of knowing what we could do and perhaps should not do.” (The name of the rose- Umberto Eco. 1932-2016. Italian semiologist, philosopher and writer). Sickle cell disease (SCD) is a worldwide distributed monogenic red cell disorder with still high morbidity, limited therapeutic options, and a negative impact on patient quality of life [1,2,3]. Recently, gene addition/gene editing based therapeutic approaches for patients with SCD have been approved by the European Medical Agency (EMA) and the food and drug administration (FDA) and ready to be available in real world despite previous concerns on insertional oncogenesis by gene insertion [4], and limitation due to the very high product cost [5, 6].