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Πλοήγηση ανά Θέμα "off-target effect"

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    • In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies 

      Li C., Georgakopoulou A., Newby G.A., Everette K.A., Nizamis E., Paschoudi K., Vlachaki E., Gil S., Anderson A.K., Koob T., Huang L., Wang H., Kiem H.-P., Liu D.R., Yannaki E., Lieber A. (2022)
      Individuals with β-thalassemia or sickle cell disease and hereditary persistence of fetal hemoglobin (HPFH) possessing 30% fetal hemoglobin (HbF) appear to be symptom free. Here, we used a nonintegrating HDAd5/35++ vector ...