Browsing by Author "Davies, J."

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Base editing repairs the hbe mutation restoring the production of normal globin chains in severe hbe/β-Thalassemia patient hematopoietic stem and erythroid cells

Badat, M.; Hua, P.; Mettananda, S.; Fisher, C.; Roy, N.; Rice, S.; Roy, A.; Higgs, D.; Davies, J. (European Hematology Association, 2022)

Aims: HbE/β-thalassemia is the commonest form of severe β-thalassemia, and comprises approximately 50% of all cases worldwide1. HbE/β-thalassemia is caused by the HbE codon 26 G>A mutation on one allele and any β0-thalassemia ...
Base editing repairs the HBE mutation restoring the production of normal globin chains in severe HBE/β-thalassemia patient hematopoietic stem and erythroid cells

Badat, M.; Hua, P.; Mettananda, S.; Fisher, C.; Roy, N.; Rice, S.; Roy, A.; Higgs, D.; Davies, J. (Elsevier, 2021)

HbE/β-thalassemia is the commonest form of severe β-thalassemia, and comprises approximately 50% of all cases worldwide. HbE/β-thalassemia is caused by the HbE codon 26 G>A mutation on one allele and any severe β 0-thalassemia ...
Genome editing of haemopoietic stem cells for treatment of thalassaemia

Badat, M.; Mettananda, S.; Hua, P.; Schwessinger, R.; Hughes, J.; Higgs, D.; Davies, J. (Oxford University Press, 2019)

AIM: Thalassaemia is commonly due to mutations at the beta globin (HBB) locus, and this causes transfusion dependent anaemia in severe cases. A key pathophysiological factor is the imbalance of alpha and beta globin ...

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