A one-base therapeutic insertion in the HBG2 distal promoter reactivates γ-globin expression
Overall, we presented the preclinical data to support the role of insA on regulating γ-globin expression using CD34<sup>+</sup> HSPC cells derived from healthy donors or patients with β-thalassemia, and subsequently engrafted mice. Our study suggests that introducing insA mutation leads to significantly boosted fetal globin levels and uncovers new safe therapeutic target or strategy for β-hemoglobinopathies.
Authors
- F. Chen
- Xiuqin Bao (ORCID: https://orcid.org/0000-0002-9935-9851)
- Liren Wang (ORCID: https://orcid.org/0000-0003-1890-6575)
- Yuhua Ye (ORCID: https://orcid.org/0000-0003-4443-3847)
- Zhongju Wang (ORCID: https://orcid.org/0000-0001-5579-4280)
- Li Du (ORCID: https://orcid.org/0000-0003-2394-0727)
- Xiangmin Xu (ORCID: https://orcid.org/0000-0003-2775-8228)
- Jing Du (ORCID: https://orcid.org/0000-0003-3128-9997)
- Xiaoyi Chen (ORCID: https://orcid.org/0000-0002-7378-5158)
- Yuanyi Gao (ORCID: https://orcid.org/0000-0001-6978-1149)
- Xiaoqin Feng (ORCID: https://orcid.org/0000-0003-1166-0138)
- Aihua Yin (ORCID: https://orcid.org/0000-0001-9498-9777)
Institutions
- Nanfang Hospital (CN)
- Guangdong Province Women and Children Hospital (CN)
- Southern Medical University (CN)
- East China Normal University (CN)
- Guangzhou Medical University (CN)
Publication Details
- Journal
- Experimental Hematology and Oncology
- Published
- 2025-03-28
- DOI
- https://doi.org/10.1186/s40164-025-00626-7
- Citations
- 1
- Primary Topic
- Hemoglobinopathies and Related Disorders
- Type
- article
- Field-Weighted Citation Impact
- 1.63
Funders
- National Natural Science Foundation of China
- Guangzhou Municipal Science and Technology Project