对失明的基因治疗可能很快就会成为现实

2017-11-12 MedSci MedSci原创

美国眼科学会2017年第121届年度会议AAO的一项研究表明，那些在遗传性视网膜疾病中丧失视力的患者在接受了一种新的基因疗法治疗后，可以很好地通过迷宫。该研究的患者有一种名为Leber先天性黑朦(LCA)的病症，该病症始于婴儿期，进展缓慢，最终导致完全失明。目前，美国食品和药物管理局正在对这一新的，首创的基因疗法进行审查，有望在今年获得批准。目前还没有治疗遗传性视网膜疾病的治疗方法。爱荷华大学的眼

美国眼科学会2017年第121届年度会议AAO的一项研究表明，那些在遗传性视网膜疾病中丧失视力的患者在接受了一种新的基因疗法治疗后，可以很好地通过迷宫。该研究的患者有一种名为Leber先天性黑朦(LCA)的病症，该病症始于婴儿期，进展缓慢，最终导致完全失明。目前，美国食品和药物管理局正在对这一新的，首创的基因疗法进行审查，有望在今年获得批准。目前还没有治疗遗传性视网膜疾病的治疗方法。爱荷华大学的眼科医生Stephen R. Russell博士是这一开创性治疗的主要研究人员之一。来自第一项随机对照的3期研究的数据显示，29例接受治疗的患者中有27例（93％）的视力有明显的改善，足以使他们能够在低至中度的光线下行走迷宫。他们还表现出对光敏感度和周边视力的改善，这是这些患者经历的两种视觉缺陷。批准可能为其他基因疗法打开大门，最终可以治疗已知的超过225种导致失明的基因突变。它可以应用于色素性视网膜炎，这是一种由基因缺陷引起的遗传性视网膜疾病。或者在未来，基因疗法可能提供关键的蛋白质，以恢复像与年龄相关的黄斑变性等更常见疾病的视力。LCA非常罕见， 8万名患者中约有1人患有这种病。它可能由19

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2017-11-13 李东泽

是很好的学习材料.不错.以后会多学习.

110 0
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2017-11-12 李东泽

是很好的学习材料.不错.以后会多学习.

104 0
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2017-11-12 明月清辉

谢谢分享.学习了

113 0
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2017-11-12 lyh994

作为携带健康基因的载体进入视网膜.医生将数十亿的改良病毒注入患者的双眼

123 0

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