Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease.
Julie KanterMark C WaltersLakshmanan KrishnamurtiMarkus Y MaparaJanet L KwiatkowskiStacey Rifkin-ZenenbergBanu AygunKimberly A KasowFrancis J PiercieyMelissa BonnerAlex MillerXinyan ZhangJessie LynchDennis KimJean-Antoine RibeilMohammed AsmalSunita GoyalAlexis A ThompsonJohn F TisdalePublished in: The New England journal of medicine (2021)
in most red cells, leading to reduced hemolysis and complete resolution of severe vaso-occlusive events. (Funded by Bluebird Bio; HGB-206 ClinicalTrials.gov number, NCT02140554.).