Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease | New England Journal of Medicine www.nejm.org/doi/full/10....
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Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease | NEJM
Exagamglogene autotemcel (exa-cel) is a cell therapy in which autologous CD34+ hematopoietic cells are engineered through ex vivo clustered regularly interspaced short palindromic repeats–Cas9 edit...