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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Bibliografiske detaljer

Principais autores:	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
Format:	Artigo
Sprog:	Inglês
Udgivet:	Elsevier 2024-06-01
Serier:	Molecular Therapy: Nucleic Acids
Fag:	MT: RNA/DNA editing CRISPR/Cas9 gene therapy hemoglobinopathies β-thalassemia near-PAMless
Online adgang:	http://www.sciencedirect.com/science/article/pii/S2162253124000702
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