Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by <i>HBB^AS3</i> Gene Addition and Mutation-Specific RNA Interference

Therapy via the gene addition of the anti-sickling β^AS3-globin transgene is potentially curative for all β-hemoglobinopathies and therefore of particular clinical and commercial interest. This study investigates GLOBE-based lentiviral vectors (LVs) for β^AS3-globin addition and...

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Autors principals:	Lola Koniali, Christina Flouri, Markela I. Kostopoulou, Nikoletta Y. Papaioannou, Panayiota L. Papasavva, Basma Naiisseh, Coralea Stephanou, Anthi Demetriadou, Maria Sitarou, Soteroula Christou, Michael N. Antoniou, Marina Kleanthous, Petros Patsali, Carsten W. Lederer
Format:	Artigo
Idioma:	Inglês
Publicat:	MDPI AG 2023-12-01
Col·lecció:	Cells
Matèries:	gene therapy RNA interference shRNAmiR lentiviral vector hemoglobinopathy thalassemia
Accés en línia:	https://www.mdpi.com/2073-4409/12/24/2848
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