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Context base editing for splice correction of IVSI-110 β-thalassemia

β-Thalassemia is brought about by defective β-globin (HBB [hemoglobin subunit β]) formation and, in severe cases, requires regular blood transfusion and iron chelation for survival. Genome editing of hematopoietic stem cells allows correction of underlying mutations as curative therapy. As potential...

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Main Authors: Basma Naiisseh (Author), Panayiota L. Papasavva (Author), Nikoletta Y. Papaioannou (Author), Marios Tomazou (Author), Lola Koniali (Author), Xenia Felekis (Author), Constantina G. Constantinou (Author), Maria Sitarou (Author), Soteroula Christou (Author), Marina Kleanthous (Author), Carsten W. Lederer (Author), Petros Patsali (Author)
Format: Book
Published: Elsevier, 2024-06-01T00:00:00Z.
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