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.
Toediening AAV5-hFVIII-SQ geassocieerd met normalisatie factor VIII-activiteit
jan 2018 | Benigne hematologie