GenomicsbioRxivPreprint
Precise modelling and correction of a spectrum of β-thalassemic mutations in human erythroid cells by base editors
K. Manjunatha Prasad, Nivedhitha Devaraju, Anila George…·Kumarasamypet Murugesan Mohankumar
This study demonstrates the use of base editors to precisely correct various β-thalassemic mutations in human erythroid cells, showing efficient and scarless mutation correction with normal hemoglobin variants.
DOI 10.1101/2022.06.01.494256