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Πλοήγηση ανά Θέμα "hemoglobin F"

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    • The 5′ regulatory region of the human fetal globin genes is a gene conversion hotspot 

      Kalamaras, A.; Chassanidis, C.; Samara, M.; Chiotoglou, I.; Vamvakopoulos, N. K.; Papadakis, M. N.; Kollia, P.; Patrinos, G. P. (2008)
      The human fetal globin genes consist of the first mammalian genomic loci for which gene conversion was reported. To date, 14 gene conversions have been described in the human Gγ- and Aγ-globin genes, the vast majority of ...

    • 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 ...