基因疗法显示对抗镰状细胞病的希望

S. Phadnis
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摘要

镰状细胞病(SCD)是一种遗传性血液疾病,其特征是存在异常血红蛋白(HbS)等位基因以及可引起这种异常分子聚合的第二等位基因。这种状态是由于基因突变使红细胞扭曲成镰刀状而产生的。镰状细胞阻止红细胞通过血管,阻止氧气的供应,从而导致贫血、高血压和器官衰竭等严重并发症
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Gene Therapy Shows Promise Against Sickle – Cell Disease
Sickle Cell Disease (SCD) is a genetic blood disorder which is characterized by presence of abnormal hemoglobin (HbS) allele along with a second allele that can cause polymerization of such abnormal molecule. The state arises due to a genetic mutation that distorts red blood cells into sickle shaped. Sickle cells block red blood cells from passing through the blood vessels and prevent the supply of oxygen that results into severe complications like anemia, hypertension and organ failure
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