Frangoul, Haydar ; Altshuler, David ; Cappellini, M. Domenica ; Chen, Yi-Shan ; Domm, Jennifer ; Eustace, Brenda K. ; Foell, Juergen ; de la Fuente, Josu ; Grupp, Stephan ; Handgretinger, Rupert ; Ho, Tony W. 
; Kattamis, Antonis ; Kernytsky, Andrew ; Lekstrom-Himes, Julie ; Li, Amanda M. ; Locatelli, Franco ; Mapara, Markus Y. ; de Montalembert, Mariane ; Rondelli, Damiano ; Sharma, Akshay ; Sheth, Sujit ; Soni, Sandeep ; Steinberg, Martin H. ; Wall, Donna ; Yen, Angela ; Corbacioglu, Selim
; Kattamis, Antonis ; Kernytsky, Andrew ; Lekstrom-Himes, Julie ; Li, Amanda M. ; Locatelli, Franco ; Mapara, Markus Y. ; de Montalembert, Mariane ; Rondelli, Damiano ; Sharma, Akshay ; Sheth, Sujit ; Soni, Sandeep ; Steinberg, Martin H. ; Wall, Donna ; Yen, Angela ; Corbacioglu, Selim 
Zusammenfassung
Transfusion-dependent beta-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is a transcription factor that represses gamma-globin expression and fetal hemoglobin in erythroid cells. We performed electroporation of CD34+ hematopoietic stem and progenitor cells obtained from healthy donors, with ...
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