受基因缺陷影响,视网膜中的感光细胞会慢慢地停止工作并死亡。麦克拉伦教授的团队决定在实验室制作健康的基因,并将每个基因都装进一个微小病毒(这种病毒并不会导致人类患病)中,然后将这种混合体注入到视网膜下面。
The therapy is given in one eye so it's easy to compare the progression of the disease with the untreated eye. 'Every injection has 10 billion viral particles, each carrying one copy of the gene,' said Prof. MacLaren. 'We have to target millions of cells.'
每个患者只有一只眼睛接受了这种疗法,因此通过未经治疗的那只眼睛,研究人员很容易就能对比病症的变化。麦克拉伦教授说,研究人员每次注入100亿个病毒颗粒,每个颗粒都携带一份基因。他说,我们需要将数以百万计的细胞作为目标。
The trial began with six patients. Two still had excellent visual acuity-clearness of vision-which is measured by reading lines of letters on a sight chart. Two other patients had good acuity and two had reduced acuity.
试验是从六位病人开始的。其中两位病人仍拥有非常高的视敏度,两位病人拥有较高的视敏度,另外两位病人有着较低的视敏度。视敏度也被称为视觉清晰度,通过让病人阅读视力测验表上的字母来衡量。
Six months after the operation, the two patients with reduced acuity showed improved vision, being able to read two and four more lines on the sight chart. The others could see better in dim light. The gains were sustained over several months of follow-up.
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