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

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

Therapy via the gene addition of the anti-sickling β<sup>AS3</sup>-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<sup>AS3</sup&...

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Bibliographic Details
Main Authors: 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: Article
Language:English
Published: MDPI AG 2023-12-01
Series:Cells
Subjects:
gene therapy
RNA interference
shRNAmiR
lentiviral vector
hemoglobinopathy
thalassemia
Online Access:https://www.mdpi.com/2073-4409/12/24/2848

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https://www.mdpi.com/2073-4409/12/24/2848

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