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Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

Authors :
Hardouin, Giulia
Antoniou, Panagiotis
Martinucci, Pierre
Felix, Tristan
Manceau, Sandra
Joseph, Laure
Masson, Cécile
Scaramuzza, Samantha
Ferrari, Giuliana
Cavazzana, Marina
Miccio, Annarita
Source :
Blood; March 2023, Vol. 141 Issue: 10 p1169-1179, 11p
Publication Year :
2023

Abstract

•Adenine base editors efficiently correct the IVS1-110 (G>A) β-thalassemia–causing mutation.•Base editing efficiently corrects the β-thalassemic phenotype in vitro and in vivo.

Details

Language :
English
ISSN :
00064971 and 15280020
Volume :
141
Issue :
10
Database :
Supplemental Index
Journal :
Blood
Publication Type :
Periodical
Accession number :
ejs61423835
Full Text :
https://doi.org/10.1182/blood.2022016629