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福克斯新闻:美国药管局批准第一种针对遗传病的基因疗法

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Advisors to the Food and Drug Administration are meeting Thursday to consider giving the go ahead to gene therapy aimed at improving vision for some people with hereditary blindness.

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It would be the first gene therapy in the U.S. for an inherited disease and the first in which a corrective gene is given directly to a patient. The eye treatment Luxturna is intended to be given just once and supplies a gene to make a protein needed for sight which is missing in people with a defective gene. A study of 29 patients found it improved vision for nearly all who received it and it seemed safe.

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Luxturna is made by Philadelphia based Spark Therapeutics.

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Lilian Woo, FOX News.

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重点单词   查看全部解释    
hereditary [hi'reditəri]

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adj. 世袭的,遗传的

 
defective [di'fektiv]

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adj. 有缺陷的,不完全变化的(动词) n. 有缺陷的

 
blindness ['blaidnis]

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n. 失明;无知;[军]盲区

 
therapy ['θerəpi]

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n. 疗法,治疗

 
intended [in'tendid]

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adj. 故意的,有意的;打算中的 n. 已订婚者 v.

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administration [əd.mini'streiʃən]

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n. 行政,管理,行政部门

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protein ['prəuti:n]

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n. 蛋白质

 
spark [spɑ:k]

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n. 火花,朝气,情人,俗丽的年轻人
vi.

 
gene [dʒi:n]

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n. 基因

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woo [wu:]

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v. 向 ... 求爱,追求,恳求

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