基因疗法有望被用于改善视力
<p><span word="Scientists">Scientists</span> <span word="have">have</span> <span word="improved">improved</span> <span word="the">the</span> <span word="vision">vision</span> <span word="of">of</span> <span word="a">a</span> <span word="small">small</span> <span word="number">number</span> <span word="of">of</span> <span word="patients">patients</span> <span word="suffering">suffering</span> <span word="from">from</span> <span word="a">a</span> <span word="rare">rare</span> <span word="and">and</span> <span word="incurable">incurable</span> <span word="eye">eye</span> <span word="disease">disease</span> <span word="by">by</span> <span word="replacing">replacing</span> <span word="a">a</span> <span word="defective">defective</span> <span word="gene">gene</span> <span word="with">with</span> <span word="a">a</span> <span word="healthy">healthy</span> <span word="one">one</span>-<span word="a">a</span> <span word="boost">boost</span> <span word="for">for</span> <span word="a">a</span> <span word="technique">technique</span> <span word="known">known</span> <span word="as">as</span> <span word="gene">gene</span> <span word="therapy">therapy</span>.</p><p>通过将有缺陷的基因替换成健康的基因，科学家已经使少数患有一种罕见、难愈眼病的病人改善了视力。这对一项被称为基因疗法的技术是个推动。</p><p><span word="The">The</span> <span word="patients">patients</span> <span word="have">have</span> <span word="choroideremia">choroideremia</span>, <span word="a">a</span> <span word="degenerative">degenerative</span> <span word="disease">disease</span> <span word="caused">caused</span> <span word="by">by</span> <span word="defects">defects</span> <span word="in">in</span> <span word="a">a</span> <span word="single">single</span> <span word="gene">gene</span> <span word="that">that</span> <span word="leads">leads</span> <span word="to">to</span> <span word="blindness">blindness</span> <span word="and">and</span> <span word="affects">affects</span> 1 <span word="in">in</span> 50,000 <span word="people">people</span>. <span word="In">In</span> <span word="an">an</span> <span word="early">early</span>-<span word="stage">stage</span> <span word="trial">trial</span> <span word="published">published</span> <span word="Wednesday">Wednesday</span> <span word="in">in</span> <span word="the">the</span> <span word="Lancet">Lancet</span>, <span word="the">the</span> <span word="researchers">researchers</span> <span word="used">used</span> <span word="a">a</span> <span word="deactivated">deactivated</span> <span word="virus">virus</span> <span word="to">to</span> <span word="safely">safely</span> <span word="ferry">ferry</span> <span word="billions">billions</span> <span word="of">of</span> <span word="healthy">healthy</span>, <span word="lab">lab</span>-<span word="made">made</span> <span word="versions">versions</span> <span word="of">of</span> <span word="the">the</span> <span word="gene">gene</span> <span word="into">into</span> <span word="the">the</span> <span word="retina">retina</span>. <span word="That">That</span> <span word="appeared">appeared</span> <span word="to">to</span> <span word="restore">restore</span> <span word="the">the</span> <span word="function">function</span> <span word="of">of</span> <span word="light">light</span>-<span word="sensitive">sensitive</span> <span word="cells">cells</span>, <span word="which">which</span> <span word="the">the</span> <span word="disease">disease</span> <span word="impairs">impairs</span>.</p><p>上述病人患有脉络膜缺失症。这是一种由单一基因缺陷造成的退化性疾病，会导致失明，每50,000人就有1例。医学期刊《柳叶刀》(<span word="Lancet">Lancet</span>)周三刊登了初步试验的结果。在试验中，研究人员使用了一种失去活性的病毒来携带实验室制作的健康基因，将数以十亿计的这类基因安全地注入视网膜。这在表观上恢复了疾病损害的感光细胞的功能。</p><p>'<span word="We">We</span> <span word="were">were</span> <span word="surprised">surprised</span> <span word="by">by</span> <span word="the">the</span> <span word="magnitude">magnitude</span> <span word="of">of</span> <span word="vision">vision</span> <span word="improvement">improvement</span>' <span word="in">in</span> <span word="the">the</span> <span word="patients">patients</span>, <span word="said">said</span> <span word="Robert">Robert</span> <span word="MacLaren">MacLaren</span>, <span word="a">a</span> <span word="professor">professor</span> <span word="of">of</span> <span word="ophthalmology">ophthalmology</span> <span word="at">at</span> <span word="the">the</span> <span word="University">University</span> <span word="of">of</span> <span word="Oxford">Oxford</span> <span word="in">in</span> <span word="England">England</span> <span word="and">and</span> <span word="leader">leader</span> <span word="of">of</span> <span word="the">the</span> <span word="clinical">clinical</span> <span word="trial">trial</span>.</p>
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