英国牛津大学(University of Oxford)眼科学教授、上述临床试验的负责人麦克拉伦(Robert MacLaren)称,病人视力的改善程度出乎意料。
The experiment marks one of the first times that gene therapy has targeted the main light-sensing cells in the retina. It thus offers a possible route for treating far more common causes of blindness that affect the same cells, such as retinitis pigmentosa and age-related macular degeneration.
这是以视网膜主要感光细胞为目标开展基因疗法的首批试验之一,由此可能给治疗影响主要感光细胞的更常见失明症(比如色素性视网膜炎和老年黄斑退化)提供一条途径。
Gene therapy involves the use of DNA, rather than a protein or drug, to treat an ailment. The idea is that if the DNA gets properly embedded in the target cells, it can potentially remain there indefinitely and deliver benefits for a long time.
基因疗法使用DNA治疗疾病,而不是使用蛋白质或药物。这种疗法的思路是,在被妥善嵌入目标细胞的情况下,DNA可能会永远留在细胞中,从而给病人带来长期的益处。
Gene therapy fell out of favor after a handful of early studies led to cancer and death. That's a big reason why it hasn't yet led to a single authorized treatment in the U.S.
基因疗法一度失去关注,因为一些早期研究曾经导致癌症和死亡。这就是美国还没有批准任何一项基因疗法的重要原因。
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