遗传增强
基因组编辑
锌指核酸酶
清脆的
胎儿血红蛋白
疾病
基因
生物
临床试验
地中海贫血
生物信息学
转录激活物样效应核酸酶
医学
Cas9
核酸酶
计算生物学
遗传学
胎儿
内科学
怀孕
作者
Franco Locatelli,Marina Cavazzana,Haydar Frangoul,Josu de la Fuente,Mattia Algeri,Roland Meisel
标识
DOI:10.1016/j.ymthe.2024.03.005
摘要
In recent years, a growing number of clinical trials have been initiated to evaluate gene therapy approaches for the treatment of patients with transfusion-dependent β-thalassemia and sickle cell disease (SCD). Therapeutic modalities being assessed in these trials utilize different molecular techniques, including lentiviral vectors to add functional copies of the gene encoding the hemoglobin β subunit in defective cells and CRISPR-Cas9, transcription activator-like effector protein nuclease, and zinc finger nuclease gene editing strategies to either directly address the underlying genetic cause of disease or induce fetal hemoglobin production by gene disruption. Here, we review the mechanisms of action of these various gene addition and gene editing approaches and describe the status of clinical trials designed to evaluate the potentially for these approaches to provide one-time functional cures to patients with transfusion-dependent β-thalassemia and SCD.
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