A one-base therapeutic insertion in the HBG2 distal promoter reactivates γ-globin expression

A one-base therapeutic insertion in the HBG2 distal promoter reactivates γ-globin expression

Abstract Background The reactivation of developmental silenced γ-globin genes (HBG1/2) has shown promise as a therapeutic strategy for improving symptoms of β-hemoglobinopathies. Currently, the focus of therapeutic targets is primarily on the major fetal hemoglobin suppressors, such as BCL11A and ZB...

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Main Authors: Xiuqin Bao, Yuanyi Gao, Xiaoyi Chen, Zhongju Wang, Xiaoqin Feng, Liren Wang, Jing Du, Yuhua Ye, Feijing Chen, Li Du, Aihua Yin, Xiangmin Xu
Format: Article
Language:English
Published: BMC 2025-03-01
Series:Experimental Hematology & Oncology
Subjects:
β-thalassemia
HBG2 distal promoter
One-base therapeutic insertion
Therapeutic target
Methylation
CD34+ HSPC cells
Online Access:https://doi.org/10.1186/s40164-025-00626-7
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https://doi.org/10.1186/s40164-025-00626-7

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