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American health officials recently approved the first gene therapy treatment for an inherited disease – the type that can pass from parent to child.
美国卫生官员最近批准了第一种治疗遗传性疾病的基因疗法。遗传性疾病是指可以从父母传给孩子的疾病。
The treatment improves the sight of patients suffering from a genetic mutation that destroys their ability to see. It is the first gene therapy approved where a corrective gene is given directly to patients.
这种疗法可以改善受基因突变破坏视觉能力的患者的视力。它是第一种获得批准的将修正基因直接赋予病人的基因疗法。
People with the disease usually start losing their sight before they are 18. This often progresses to total blindness. The gene that causes the disease could suddenly appear when a child is born with a copy of the gene from both parents.
患有这种疾病的患者通常会在18岁之前丧失视力，这往往会导致完全失明。当孩子遗传了来自父母双方的这种致病基因时，这种基因可能会突然呈现。
Only a few thousand people in the U.S. are thought to have the condition.
美国只有几千人被认为患有这种疾病。
Patients receive the therapy, called Luxturna, through two injections, one for each eye. These replace the problem gene that prevents the retina, a tissue at the back of the eye, from changing light into electronic signals sent to the brain.
患者通过两次注射，每只眼睛接受一次注射来接受这种被称为Luxturna的疗法。这些基因会取代妨碍视网膜将光线转化为电子信号发送给大脑的问题基因。视网膜是眼睛里面的一种组织。
Mistie Lovelace received one of the early treatments. She said, "One of the best things I've ever seen since surgery are the stars. I never knew that they were little dots that twinkled."
米斯提·洛夫雷斯接受了这种早期治疗。她说：“手术之后我看到的最美好的东西就是星星。我从来不知道它们就是些闪烁的小光点。”
Drugmaker Spark Therapeutics has not set the price for the treatment. Its own research, however, has put the value of the therapy at around $1 million.
制药商Drugmaker尚未确定这种疗法的价格。然而，该公司自己的研究将这种疗法的价值定在1百万美元左右。
The company says the reason for the high price is due to their prediction that Luxturna will be given only once, with lasting positive effects. To date, the company has researched patients in a study for as long as four years and has not seen their vision worsen.
该公司表示，高价格是因为他们预测Luxturna疗法只有一次能够具有持久的积极影响。迄今为止，该公司已经对病人进行了长达四年的研究，并且他们的视力没有出现恶化。
The Food and Drug Administration has approved three gene therapies since August. The two other treatments are specially designed treatments for forms of blood cancer.
自8月份以来，美国食品和药品管理局已经批准了三种基因疗法。另两种疗法是对白血病的特别定制疗法。
