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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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Autori principali: Basma Naiisseh, Panayiota L. Papasavva, Nikoletta Y. Papaioannou, Marios Tomazou, Lola Koniali, Xenia Felekis, Constantina G. Constantinou, Maria Sitarou, Soteroula Christou, Marina Kleanthous, Carsten W. Lederer, Petros Patsali
Natura: Artigo
Lingua:Inglês
Pubblicazione: Elsevier 2024-06-01
Serie:Molecular Therapy: Nucleic Acids
Soggetti:
MT: RNA/DNA editing
CRISPR/Cas9
gene therapy
hemoglobinopathies
β-thalassemia
near-PAMless
Accesso online:http://www.sciencedirect.com/science/article/pii/S2162253124000702
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