Improvement and decline in vision with gene therapy in childhood blindness.
Improvement and decline in vision with gene therapy in childhood blindness.
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DOI：
10.1056/nejmoa1412965
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发表时间：
2015-05-14
期刊：
影响因子：
--
通讯作者：
Hauswirth WW
中科院分区：
文献类型：
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作者：
Jacobson SG;Cideciyan AV;Roman AJ;Sumaroka A;Schwartz SB;Heon E;Hauswirth WW
莱伯先天性黑蒙（一种常染色体隐性遗传性儿童失明）的视网膜基因治疗被广泛认为是安全有效的。治疗三年后，视力仍保持改善，但接受治疗的视网膜中光感受器的损失率与未经治疗的视网膜相同。在这里，我们描述了三名接受治疗的患者的长期随访数据。其中两名患者治疗后近 6 年，第三名患者治疗后 4.5 年，治疗区域的视觉敏感性地形图表明视力改善区域逐渐缩小。
Retinal gene therapy for Leber’s congenital amaurosis, an autosomal recessive childhood blindness, has been widely considered to be safe and efficacious. Three years after therapy, improvement in vision was maintained, but the rate of loss of photoreceptors in the treated retina was the same as that in the untreated retina. Here we describe long-term follow-up data from three treated patients. Topographic maps of visual sensitivity in treated regions, nearly 6 years after therapy for two of the patients and 4.5 years after therapy for the third patient, indicate progressive diminution of the areas of improved vision.