Now, the technique is making a comeback. For example, in three early-stage clinical trials done a few years ago, the technique was used against a retinal disease called Leber congenital amaurosis. And in November 2017, European regulators approved a gene therapy treatment for a rare condition that leaves patients unable to properly digest fats-the first such approval in the Western world.
现在这种技术正在重获关注。例如,在几年前完成的三项早期临床试验中,基因疗法技术被用于治疗一种名叫莱伯氏先天性黑蒙症的视网膜疾病。另一个例子是在2017年11月,欧洲监管机构批准用一项基因疗法治疗一种导致病人不能有效消化脂肪的罕见症状,开启了西方世界批准基因疗法的先例。
The ailment treated in the Lancet study, choroideremia, is caused by defects in a single gene on the X chromosome and mainly affects boys. Many start losing night vision by age 10 and become legally blind in their 40s.
《柳叶刀》上发表的研究治疗的脉络膜缺失症是由X染色体的单一基因缺陷引起的,主要患者为男孩。许多患者在10岁时开始失去夜视力,在40岁-50岁时变得彻底失明。
Because of the defective gene, light-sensitive cells in the retina slowly stop working and then die. Prof. MacLaren's team decided to make healthy versions of the gene in the lab, load each onto a small virus (one that doesn't cause disease in people) and inject the mix under the retina.
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