美国批准一种罕见失明症的基因疗法 分享到
Gene Therapy for Rare Form of Blindness Approved in US 2017-12-2757310

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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.
美国卫生官员最近首次批准了一种治疗遗传性疾病的基因疗法。遗传性疾病是指父母可以遗传给孩子的疾病。
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破破坏视觉能力的患者的势力。它是第一种获得批准的将修正基因直接给予病人的基因疗法。
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岁前丧失视力,通常会逐渐完全失明。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显现。
Only a few thousand people in the U.S. are thought to have the condition.
在美国,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.
患者接受一种被称为Luxturna的疗法,每只眼睛各接受一次注射。这些基因会取代问题基因,问题基因会妨碍视网膜(眼睛里的一种组织)将光线转化为电子信号发送给大脑。
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·罗夫雷斯接早期治疗的一种。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.
制药商斯帕克医疗公司尚未确定这种疗法的价格。然而,该公司自己的研究将其定价定在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,高价格是因为他们估计Luxturna疗法只需一次,且具有持久积极影响。迄今为止,该公司已经对病人进行了长达四年的研究,这些病人的视力并没有出现恶化。
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。
The approval marks an important growth for a field of study that has struggled with safety concerns.
获得批准标志着与安全问题斗争的研究领域取得重要发展。
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,一名在宾夕法尼亚大学接受罕见代谢紊乱疾病治疗的患者去世。在另一起病例中,接受一种免疫疾病治疗的患者后来换上了白血病。
Dr. David Valle said the excitement about the possibilities for genetic medicine create opportunities for research on individual diseases.
戴维·瓦勒博士表示,遗传医学的这种可能性为研究具体疾病创造了机会。
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.
宾夕法尼亚大学研究员吉恩·班纳特博士表示,她和她的丈夫伯特·马奎尔博士最初在上世纪80年年代中期就设想了利用遗传医学治疗视网膜疾病导致的失明。
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.“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.
但是开发这类科学技术花了很多年时间。第一次动物试验是在2000年,第一次人体试验是在2007年。班纳特表示:“我们不知道是什么基因导致了这种疾病,我们不具备这些基因的动物模型,我们不具备克隆基因并将它传递到视网膜的能力,所以需要花费时间来开发以上这些技术。”
I’m Phil Dierking.
菲尔·德尔金为您报道。

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