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Anne Chalumeau

inserm umr1163
H指数:11
被引数:544

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A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease

一种用于重写γ-球蛋白启动子并重新激活胎儿血红蛋白以治疗镰状细胞病的碱基编辑策略

Blood
IF23.1
2025-08-280
OAAI
Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease

严重炎症和谱系偏移与 sickle cell disease 患者中工程化造血干细胞的植入不良相关。

2025-04-010
OA
Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells
2024-06-113
OAAI
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies

结合基因添加和基因沉默策略的镰状细胞病基因治疗的新型慢病毒载体

2023-06-015
OAAI
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

Γ-珠蛋白顺式调节元件的碱基编辑介导的解剖,用于胎儿血红蛋白表达的治疗性再激活

2022-11-0434
OAAI
Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial
2022-01-2463
PRE
Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease

慢病毒与基因组编辑技术结合治疗镰状细胞病

2022-01-016
OA
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype
2020-02-14108
OAAI