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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Hlavní autoři:	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
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	MDPI AG 2023-12-01
Edice:	Cells
Témata:	gene therapy RNA interference shRNAmiR lentiviral vector hemoglobinopathy thalassemia
On-line přístup:	https://www.mdpi.com/2073-4409/12/24/2848
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