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Πλοήγηση ανά Θέμα "hemoglobin gamma chain"

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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 ...

    • Success Stories and Challenges Ahead in Hematopoietic Stem Cell Gene Therapy: Hemoglobinopathies as Disease Models 

      Yannaki E., Psatha N., Papadopoulou A., Athanasopoulos T., Gravanis A., Roubelakis M.G., Tsirigotis P., Anagnostopoulos A., Anagnou N.P., Vassilopoulos G. (2021)
      Gene therapy is a relatively novel field that amounts to around four decades of continuous growth with its good and bad moments. Currently, the field has entered the clinical arena with the ambition to fulfil its promises ...