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生物通
11 小时
科学家们发现了一种治疗镰状细胞病的潜在新基因变异靶点
FLT1是科学家从3751名镰状细胞病患者的GWAS数据中发现的14个新的胎儿血红蛋白遗传标记之一。胎儿血红蛋白通过静脉和动脉运送氧气,但在出生后不久就被成人血红蛋白所取代。镰状细胞病只影响成人血红蛋白,使其凝结并使红细胞扭曲成镰状。旺卡姆说,通过基因编辑将出生后的胎儿血红蛋白保持在8%以上的水平,是拯救更多镰状细胞病患者的一个关键、可行的方法。
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