美國批准第一種針對遺傳病的基因療法
American health officials recently approved the first gene therapy treatment for an inherited disease – the type that can pass from parent to child.
美國衛生官員最近批准了第一種治療遺傳性疾病的基因療法。遺傳性疾病是指可以從父母傳給孩子的疾病。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.
這種療法可以改善受基因突變破壞視覺能力的患者的視力。它是第一種獲得批准的將修正基因直接賦予病人的基因療法。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.
患有這種疾病的患者通常會在18歲之前喪失視力，這往往會導致完全失明。當孩子遺傳了來自父母雙方的這種致病基因時，這種基因可能會突然呈現。
Only a few thousand people in the U.S. are thought to have the condition.
美國只有幾千人被認爲患有這種疾病。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.
患者通過兩次注射，每隻眼睛接受一次注射來接受這種被稱爲Luxturna的療法。這些基因會取代妨礙視網膜將光線轉化爲電子信號發送給大腦的問題基因。視網膜是眼睛裏面的一種組織。
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·洛夫雷斯接受了這種早期治療。她說：“手術之後我看到的最美好的東西就是星星。我從來不知道它們就是些閃爍的小光點。”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.
製藥商Drugmaker尚未確定這種療法的價格。然而，該公司自己的研究將這種療法的價值定在1百萬美元左右。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.
該公司表示，高價格是因爲他們預測Luxturna療法只有一次能夠具有持久的積極影響。迄今爲止，該公司已經對病人進行了長達四年的研究，並且他們的視力沒有出現惡化。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月份以來，美國食品和藥品管理局已經批准了三種基因療法。另兩種療法是對白血病的特別定製療法。
