VOA慢速英語：美國批準(zhǔn)一種罕見失明疾病的基因療法

Gene Therapy for Rare Form of Blindness Approved in US

美國批準(zhǔn)一種罕見失明疾病的基因療法

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(wèi)生官員最近批準(zhǔn)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。它是第一種獲得批準(zhǔn)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(dǎo)致完全失明。當(dāng)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(xiàn)。

Only a few thousand people in the U.S. are thought to have the condition.

美國只有幾千人被認為患有這種疾病。

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，每只眼睛接受一次注射來接受這種被稱為Luxturna的療法。這些基因會取代妨礙視網(wǎng)膜將光線轉(zhuǎn)化為電子信號發(fā)送給大腦的問題基因。視網(wǎng)膜是眼睛里面的一種組織。

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(Mistie Lovelace)接受了這種早期治療。她說：“手術(shù)之后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.

制藥商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，高價格是因為他們預(yù)測Luxturna療法只有一次能夠具有持久的積極影響。迄今為止，該公司已經(jīng)對病人進行了長達四年的研究，并且他們的視力沒有出現(xiàn)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(jīng)批準(zhǔn)了三種基因療法。另兩種療法是對白血病的特別定制療法。

New success for difficult field

高難度領(lǐng)域的新成功

The approval marks an important growth for a field of study that has struggled with safety concerns.

獲得批準(zhǔn)標(biāo)志著深受安全擔(dān)憂困擾的這一研究領(lǐng)域取得了重要進展 。

In 1999, a patient treated for a rare metabolic disorder at the University of Pennsylvania died. In another case, patients treated for an immune disorder later developed leukemia.

1999年，一名在賓夕法尼亞大學(xué)接受罕見代謝紊亂疾病治療的患者去世。在另一起案例中，接受一種免疫疾病治療的患者后來患上了白血病。

Dr. David Valle said the excitement about the possibilities for genetic medicine create opportunities for research on individual diseases.

戴維·瓦勒博士(Dr. David Valle)表示，遺傳醫(yī)學(xué)的這種可能性為研究具體疾病創(chuàng)造了機會。

University of Pennsylvania researcher Dr. Jean Bennett said she and her husband, Dr. Albert Maguire, first imagined using genetic medicine to treat retinal blindness in the mid-1980s.

賓夕法尼亞大學(xué)研究員吉恩·班納特博士(Dr. Jean Bennett)表示，她和她的丈夫艾伯特·馬奎爾博士(Dr. Albert Maguire)最初在上世紀80年代中期就設(shè)想了利用遺傳醫(yī)學(xué)治療視網(wǎng)膜疾病導(dǎo)致的失明。

But it took many years to develop the science and technology. The first animal tests happened in 2000 and the first human tests in 2007.

但是開發(fā)這類科學(xué)技術(shù)花了很多年時間。第一次動物試驗是在2000年，第一次人體試驗是在2007年。

"We didn't know what genes caused the disease, we didn't have animal models with those genes, we didn't have the ability to clone genes and deliver them to the retina — so it took time to develop all that," said Bennett.

班納特表示：“我們不知道是什么基因?qū)е铝诉@種疾病，我們不具備這些基因的動物模型，我們不具備克隆基因并將它傳遞到視網(wǎng)膜的能力，所以需要花費時間來開發(fā)以上這些技術(shù)。”

I'm Phil Dierking.

菲爾·德爾金報道。