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Paolo Melillo¹, Francesco Testa¹, Valentina Di Iorio¹

¹Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, University of Campania "Luigi Vanvitelli", Naples, Italy.

Ophthalmology. Retina

|January 31, 2024

概括

在患有遗传性视网膜变症的患者中,Voretigene neparvovec (VN) 治疗显著改善了视力和视网膜功能. 固定稳定性和瞳孔测量等客观测量证实了这些积极的结果超过12个月.

科学领域:

眼科医生眼科眼科
遗传学是一个遗传学.
视网膜疾病视网膜疾病

背景情况:

遗传性视网膜发育不良 (IRDs) 是一组导致渐进性视力丧失的遗传性疾病.
与RPE65相关的IRD是一种特定的亚型,可以通过基因疗法治疗.
沃雷基因 neparvovec (VN) 是一种已批准的基因疗法用于与RPE65相关的IRDs.

研究的目的:

在临床环境中评估voretigene neparvovec (VN) 治疗的疗效.
用固定稳定性和色色瞳孔测量的客观测量来评估治疗结果.
为了将VN疗效与标准视觉功能测试进行比较.

主要方法:

对12名患者 (7-34岁) 进行了回顾性队列研究,这些患者患有与RPE65相关的IRD.
患者接受了双边VN治疗,并进行了12个月的随访.
使用最佳校正视敏度 (BCVA),全场刺激值 (FST),半自动运动视野 (SKVF),微观测量和色学瞳孔测量进行评估.

主要成果:

经过45天,BCVA,SKVF和FST的显著改善被观察到,并在12个月内保持.
固定稳定性和染色瞳孔测量显示治疗后显著改善.
增加的瞳孔收缩与更高的视网膜敏感性相关,表明治疗的有效性.

结论:

在与RPE65相关的IRD中,VN治疗在改善视网膜功能方面表现出显著的有效性.
像固定稳定性和色谱瞳孔测量等客观测量对于评估治疗结果非常有价值.
染色瞳孔测量显示出作为监测基因治疗有效性的关键客观测试的希望.

关键词:

染色瞳孔测量仪的使用基因治疗是一种基因疗法.遗传性视网膜损伤遗传性视网膜损伤在 RPE65 基因中,在Voretigene neparvovec中使用.

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