Direct correction of haemoglobin E β-thalassaemia using base editors
Haemoglobin E (HbE) β-thalassaemia causes approximately 50% of all severe thalassaemia worldwide; equating to around 30,000 births per year. HbE β-thalassaemia is due to a point mutation in codon 26 of the human HBB gene on one allele (GAG; glutamatic acid → AAG; lysine, E26K), and any mutation caus...
Main Authors: | Badat, M, Ejaz, A, Hua, P, Rice, S, Zhang, W, Hentges, LD, Fisher, CA, Denny, N, Schwessinger, R, Yasara, N, Roy, NBA, Issa, F, Roy, A, Telfer, P, Hughes, J, Mettananda, S, Higgs, DR, Davies, JOJ |
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Format: | Journal article |
Language: | English |
Published: |
Springer Nature
2023
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