Interviewer: Conditions based on a single gene defect.

主持：只要一个基因有问题，就可能出现这些病症。

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Only five children born in the U.S. annually have the same type of LCA as Morehouse, but focusing on a rare single-gene defect is a good way to develop a model for treating more common ailments.

在美国出生的孩子中，每年只有5个孩子的雷伯氏先天性黑内障与豪斯为同一类型，但是从一个少见的单基因缺陷入手，是开发更普遍的治疗模型的一个很好的途径。

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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

耶鲁公开课 - 生物医学工程探索课程节选

Because of this, because cystic fibrosis is a disease that's caused by a single gene defect, there's been great interest in trying to treat cystic fibrosis with gene therapy.

就是因为如此,就因为囊胞性纤维症,是一种单基因病,人们对于用基因治疗,治愈囊胞性纤维症产生了很大的兴趣

耶鲁公开课 - 生物医学工程探索课程节选

Usually this is a disease that's caused by a defect in a single gene.

通常在疾病是由,单基因缺陷引起时才可行

耶鲁公开课 - 生物医学工程探索课程节选