Lin M, Paik E, Mishra B, et al. Re-creating hereditary persistence of fetal hemoglobin (PHFH) with CRISPR/CAS9 to treat sickle cell disease (SCD) and beta-thalassemia (beta-thal). EHA 2017, abstract S147.
Eerste gentherapie voor behandeling van ernstige hemofilie A
jul 2022 | Benigne hematologie