busulfan
otsuka
['bet', 'busulfan', 'crispr']
crispr/cas9 editing of erythroid-specific promoter of bcl11a = reversal of gamma to beta globin chain switch = high hgbf synthesis = nearly 100% cure of both transfusion-dependent thalassemia and severe sickle cell disease. incredible
#ash22 
Tweeting about transfusion medicine, clinical hematology, and anyone trying to leave the world a little better than they found it