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（在线收听）

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.

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.

Luxturna is made by Philadelphia based Spark Therapeutics.

Lilian Woo, FOX News.

周四，美国食品和药物管理局的顾问召开会议，商讨批准用于恢复遗传失明患者视力的基因治疗方案。

该方案为美国首个针对遗传病的基因治疗方案，同时也是首个直接向患者注射基因正常拷贝的治疗方案。该方案名为“Luxturna基因疗法”，只需注射一次，就能给有基因缺陷的患者提供转换蛋白质所需的基因。有研究表明，受试的29名患者的视力几乎都有了改善，该方案似安全可靠。

研发该疗法的是费城的火花基因疗法公司。

福克斯新闻，我是Lilian Woo。