Πλοήγηση ανά Θέμα "Hemoglobinopathies"
Αποτελέσματα 1-3 από 3
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In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies
(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 ... -
Molecular diagnosis of inherited disorders: Lessons from hemoglobinopathies
(2005)Hemoglobinopathies constitute a major health problem worldwide, with a high carrier frequency, particularly in certain regions where malaria has been endemic. These disorders are characterized by a vast clinical and ... -
Success Stories and Challenges Ahead in Hematopoietic Stem Cell Gene Therapy: Hemoglobinopathies as Disease Models
(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 ...