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Short-hairpin RNA against aberrant HBBIVSI-110(G>A) mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells

Department of Molecular Genetics Thalassaemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus;Department of Medical and Molecular Genetics, King’s College London, UK
Department of Molecular Genetics Thalassaemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus;Cyprus School of Molecular Medicine, Nicosia, Cyprus
Department of Molecular Genetics Thalassaemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus;Department of Medical and Molecular Genetics, King’s College London, UK
Thalassaemia Centre, Ministry of Health, Cyprus
Thalassaemia Centre, Ministry of Health, Cyprus
Department of Medical and Molecular Genetics, King’s College London, UK
Department of Molecular Genetics Thalassaemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus;Cyprus School of Molecular Medicine, Nicosia, Cyprus
Department of Molecular Genetics Thalassaemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus;Cyprus School of Molecular Medicine, Nicosia, Cyprus
Vol. 103 No. 9 (2018): September, 2018 https://doi.org/10.3324/haematol.2018.189357