來源于The Economist August 27th 2022 Leaders “Briefing Genetic medicine --- The trials of gene therapy” 這篇文章的首、二、三段。

The Economist August 27th 2022 Leaders

In 2018, when he was 13, Ethan Ralston’s eyesight started to get blurry. The diagnosis was devastating. He had been born with Leber Hereditary Optic Neuropathy (LHON), a rare genetic disorder that eats away at the cells of the optic nerve until it causes blindness.

2018年，伊森·拉爾斯頓(Ethan Ralston)13歲，他的視力開始變得模糊。診斷結果是毀滅性的。他出生時就患有Leber遺傳性視神經病變(LHON)，這是一種罕見的的遺傳性疾病，該病會損害視神經細胞，直至導緻失明。

Given that America and Europe between them see just 800 cases of LHON a year young Mr Ralston was very unlucky. In another way, though, he could be counted fortunate. GenSight, a French biotech company, had for years been working on a gene therapy for LHON. The condition is caused by a mutation in a gene called ND4 which causes the body's cells to make a faulty protein. The therapy, called Lumevoq, sought to resolve the problem by adding the canonical version of ND4 to cells in the retina and optic nerve. By 2018 Lumevoq was in clinical trials. Shortly after his diagnosis Mr Ralston was treated with it.

鑒于美國和歐洲每年僅有800例LHON患者，年輕的拉爾斯頓非常不走運。不過，從另一個角度來看，他也算是幸運的。法國生物技術公司GenSight多年來一直緻力于LHON的基因治療。LHON是由一個叫做ND4的基因突變引起，該基因突變導緻身體的細胞産生一種有缺陷的蛋白質。這種名為Lumevoq的療法試圖通過向視網膜和視神經的細胞添加ND4的标準版本來解決這個問題。到2018年，Lumevoq已進入臨床試驗階段。确診後不久，拉爾斯頓就接受了這種治療。

Today his eyesight has almost returned to normal. He can work on a computer, drive a car, go bowling with his friends. He would seem to be cured.

如今，他的視力幾乎恢複了正常。他可以在電腦上工作、開車、和朋友一起去打保齡球。他似乎已痊愈了。

note:

Leber遺傳性視神經病變是眼科比較少見的一類先天性疾病，為視神經退行性變的母系遺傳性疾病。男性患者居多，常于15～35歲發病，臨床主要表現為雙眼同時或先後急性或亞急性無痛性視力減退，同時可伴有中心視野缺失及色覺障礙。視力損害嚴重程度差異較大，可由完全正常、輕度、中度到重度。

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