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, Nithin Sam Ravi, Gokulnath Mahalingam, Vignesh Rajendiran, Lokesh Panigrahi, Vigneshwaran Venkatesan, Kartik Lakhotiya, Yogapriya Moorthy, Aswin Anand Pai, Yukio Nakamura, Ryo Kurita, Poonkuzhali Balasubramanian, Saravanabhavan Thangavel, , 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.
bioRxiv · Jun 1, 2022 · PreprintGenomics