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Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by HBBAS3 Gene Addition and Mutation-Specific RNA Interference.

Cells - 15 Dec 2023

Koniali Lola, Flouri Christina, Kostopoulou Markela I, Papaioannou Nikoletta Y, Papasavva Panayiota L, Naiisseh Basma, Stephanou Coralea, Demetriadou Anthi, Sitarou Maria, Christou Soteroula, Antoniou Michael N, Kleanthous Marina, Patsali Petros, Lederer Carsten W

Abstract excerpt

Therapy via the gene addition of the anti-sickling βAS3-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 βAS3-globin addition and evaluates strategies for an increased β-like globin expression without vector dose escalation. First, we report the development of a...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 38132168
DOI: 10.3390/cells12242848
PMCID: PMC10741507

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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