The disease is caused by a genetic defect that leads to bone marrow failure and a greater risk of cancers, such as leukaemia.

范康尼贫血症是由遗传缺陷引发的疾病，会导致骨髓功能衰竭，甚至更容易罹患癌症，例如血癌。

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He then corrected the sickle-cell genetic defect and prodded the iPS cells to develop into the type of marrow stem cell that manufactures a mouse's blood cells.

接着，他修正了这些iPS细胞的镰刀形贫血遗传缺陷，并刺激这些iPS细胞分化发育成为能够制造小鼠红细胞的骨髓干细胞。

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Andrews argues that fixing a genetic defect that causes a catastrophic disease and passing that correction to new generations would be a benefit, not a risk.

安德鲁斯争辩说，修复导致致命疾病的基因缺陷并将治疗后的基因传给下一代是一件有益的事情，而不是风险。

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It's a single gene defect and we know exactly where the genetic defect is in sickle cell.

是一种单基因缺陷,而且我们准确地知道,镰状红细胞贫血的基因缺陷在哪里

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