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

• المؤلفون الرئيسيون: Basma Naiisseh (مؤلف), Panayiota L. Papasavva (مؤلف), Nikoletta Y. Papaioannou (مؤلف), Marios Tomazou (مؤلف), Lola Koniali (مؤلف), Xenia Felekis (مؤلف), Constantina G. Constantinou (مؤلف), Maria Sitarou (مؤلف), Soteroula Christou (مؤلف), Marina Kleanthous (مؤلف), Carsten W. Lederer (مؤلف), Petros Patsali (مؤلف)
• التنسيق: كتاب
• منشور في: Elsevier, 2024-06-01T00:00:00Z.
• الموضوعات: article
• الوصول للمادة أونلاين: Connect to this object online.

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...