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科學家將要用基因編輯技術治療先天失明

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CRISPR is coming for your body. While that may sound like the tagline of a horror film, it's potentially the future of medicine, as the gene-editing technique is about to be used for the first time inside the bodies of presumably willing patients.

CRISPR(成簇規律間隔的短迴文重複序列,近年來被認爲是原核生物適應性免疫結構)即將進入你的身體。雖然這聽上去可能有點像一部恐怖電影的口號,但這也許是醫藥研究的未來,因爲基因編輯技術將首次被用於想必是自願接受的病人的身體上。

The first trial could be an impressive feat of modern medicine: Scientists hope to use CRISPR's gene-editing magic to restore sight to people with an inherited form of blindness called Leber congenital amaurosis.

這次嘗試可能是現代醫藥的一次令人印象深刻的壯舉:科學家希望利用CRISPR基因編輯的魔力來恢復遺傳性失明(又被稱爲萊伯士先天性黑朦)的人的視力。

It's the most common cause of inherited childhood blindness, occurring in about 2 to 3 of every 100,000 births, according to the AP.

據美國聯合通訊社報道,這是兒童遺傳性失明最常見的原因,每十萬個新生兒中就會有2~3個人患有遺傳性失明。

People with the disease lack just one gene, the gene that converts light into signals to the brain and lets the eyes do their job.

患有這種疾病的人只缺少一種基因,這種基因把光轉化成信號並傳入大腦,然後使眼睛發揮作用。

The hope is that adding that gene through a one-time CRISPR treatment will cure them, permanently editing their DNA and letting them see.

希望通過一次CRISPR治療就可以植入這種基因,並治癒這種疾病,使DNA被永久性編輯,讓他們得見光明。

科學家將要用基因編輯技術治療先天失明

This is undoubtedly a more noble use of CRISPR than making spicy tomatoes.

比起製造辣味的西紅柿,這無疑是CRISPR技術的一項更崇高的運用。

It's a good trial for the cutting-edge—and controversial—technology, because there's already some solid proof that the disease is treatable on a genetic level.

對於這項先進且有爭議的技術而言,這是一次不錯的嘗試,因爲已有可靠的證據表明這種疾病在基因層面上是可治療的。

Thanks to a gene therapy called Luxturna, which is already on the market, scientists know that injecting a replacement gene into cells in the retina can cure the ailment .

多虧已經上市的Luxturna基因療法,科學家瞭解到向視網膜中的細胞注入一種替代基因可以治癒疾病。

CRISPR would work by similarly delivering new genetic material to the eye, giving researchers the perfect testing ground for using the new medical procedure in humans, before turning to more complicated diseases like cancer.

CRISPR同樣通過把新的遺傳物質植入眼睛來發揮作用,這在轉向用其研究治療癌症這種更復雜的疾病之前,爲研究人員在人類身上使用這種新的醫療程序提供了完美的試驗依據。

Two companies, Editas Medicine and Allergan, will test the technique in up to 18 people at hospitals around the United States, starting this fall.

在美國周圍的醫院裏,艾迪塔斯醫藥公司與阿勒根兩家公司將於今年秋天開始在多達18人身上測試這種技術。

The new study will test children at least 3 years old and adults with a range of vision.

這項新研究將在視力情況不同的三歲以上的兒童及成年人身上進行測試。