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Base editing shows potential superiority for

Adenosine base editing restarted fetal hemoglobin expression in cells from patients with sickle cell disease. Find how St. Jude & Broad are advancing to a cure.

United statesYong chengYoonjeong jangJingjing chenYu yaoRachel levineSenthil velan bhoopalanRuopeng fengMitchell weissDavid liuJohn tisdaleYichao liShondra pruett millerAkshay sharmaHenry bellThiyagaraj mayuranathan

Base editing shows promise for treating sickle cell disease and beta thalassemia

Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease (SCD) and beta thalassemia.

United statesLily ramseySenthil velan bhoopalanCicera lazzarottoGuolian kangYong chengPhillip doerflerErin dempseyMitchell weissGregory newbyRuopeng fengYu yaoRachel levineJingjing zhangShengdar tsaiHenry bell

Precise genome editing approach can correct mutation that causes sickle cell disease

Scientists at St. Jude Children’s Research Hospital and the Broad Institute of MIT and Harvard showed how prime editing can correct the mutation that causes sickle cell disease in a potentially curative approach.

Rachel levineGregory newbyKelcee everetteR mitchell weissYong chengJohn tisdaleDavid liuKalin mayberryAndrew nelsonJessie davisYichao liAlexander sousaXiong liuPeter chenYoonjeong jangRichard merkin

Prime Editing Demonstrates Concept for Treating Sickle Cell Disease

Prime Editing Demonstrates Concept for Treating Sickle Cell Disease
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Alexander sousaDavid liuGregory newbyRachel levineErin dempseyMitchell weissKelcee everetteJessie davisThiyagaraj mayuranathanYong chengYichao liYoonjeong jangRichard merkinJohn tisdaleAndrew nelsonPeter chen