Re­ly­ing on an ul­tra-rare vari­ant, David Liu un­veils a new ap­proach to edit­ing sick­le cell

There are now at least five dif­fer­ent ap­proach­es to cur­ing sick­le cell in or near­ing hu­man test­ing from at least eight dif­fer­ent com­pa­nies or aca­d­e­m­ic cen­ters. But re­searchers have not stopped look­ing for im­prove­ments.

David Liu, the co-in­ven­tor of base edit­ing and co-founder of Beam Ther­a­peu­tics, un­veiled in Na­ture Tues­day a new ap­proach for us­ing gene edit­ing to turn pa­tients’ sick­ling he­mo­glo­bin in­to a healthy form of the pro­tein. If it plays out in hu­mans, ex­perts say, the strat­e­gy could of­fer a more di­rect and po­ten­tial­ly safer way of treat­ing the de­bil­i­tat­ing ge­net­ic dis­ease.

To read Endpoints News become a free subscriber

Unlock this article instantly, along with access to limited free monthly articles and our suite of newsletters

SIGN UP LOG IN