科學(xué)家將要用基因編輯技術(shù)治療先天失明
作者:Fast Company
2019-08-06 17:39
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(guī)律間隔的短回文重復(fù)序列,近年來被認(rèn)為是原核生物適應(yīng)性免疫結(jié)構(gòu))即將進(jìn)入你的身體。雖然這聽上去可能有點(diǎn)像一部恐怖電影的口號(hào),但這也許是醫(yī)藥研究的未來,因?yàn)榛蚓庉嫾夹g(shù)將首次被用于想必是自愿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(xiàn)代醫(yī)藥的一次令人印象深刻的壯舉:科學(xué)家希望利用CRISPR基因編輯的魔力來恢復(fù)遺傳性失明(又被稱為萊伯士先天性黑朦)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(jù)美國聯(lián)合通訊社報(bào)道,這是兒童遺傳性失明最常見的原因,每十萬個(gè)新生兒中就會(huì)有2~3個(gè)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(zhuǎn)化成信號(hào)并傳入大腦,然后使眼睛發(fā)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,并治愈這種疾病,使DNA被永久性編輯,讓他們得見光明。
This is undoubtedly a more noble use of CRISPR than making spicy tomatoes.
比起制造辣味的西紅柿,這無疑是CRISPR技術(shù)的一項(xiàng)更崇高的運(yùn)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(duì)于這項(xiàng)先進(jìn)且有爭議的技術(shù)而言,這是一次不錯(cuò)的嘗試,因?yàn)橐延锌煽康淖C據(jù)表明這種疾病在基因?qū)用嫔鲜强芍委煹摹?/div>
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(jīng)上市的Luxturna基因療法,科學(xué)家了解到向視網(wǎng)膜中的細(xì)胞注入一種替代基因可以治愈疾病。
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(zhì)植入眼睛來發(fā)揮作用,這在轉(zhuǎn)向用其研究治療癌癥這種更復(fù)雜的疾病之前,為研究人員在人類身上使用這種新的醫(yī)療程序提供了完美的試驗(yàn)依據(jù)。
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(yī)院里,艾迪塔斯醫(yī)藥公司與阿勒根兩家公司將于今年秋天開始在多達(dá)18人身上測(cè)試這種技術(shù)。
The new study will test children at least 3 years old and adults with a range of vision.
這項(xiàng)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(jìn)行測(cè)試。
(翻譯: Dlacus)
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