PDF(Pubmed)
Abstract：
Genetic pediatric eye disease frequently leads to severe vision impairment or blindness. Voretigene neparvovec is the first approved gene therapy for an inherited retinal dystrophy (IRD). Voretigene neparvovec has been shown to be well tolerated and safe, with encouraging results in terms of efficacy, mainly when administered early in childhood. While we assisted at the first gene therapy available in clinical practice for an IRD, some questions remain unanswered, especially when gene therapy is delivered in young children. We review here the most recent reports and promising ongoing studies concerning various approaches on gene therapy in pediatric ophthalmology.
摘要：
遗传性儿科眼病经常导致严重的视力障碍或失明。Voretigeneneparvovec是第一个被批准的遗传性视网膜营养不良（IRD）的基因疗法。Voretigeneneparvovec已被证明具有良好的耐受性和安全性，在疗效方面取得了令人鼓舞的结果，主要是在儿童早期服用。虽然我们为IRD的临床实践提供了第一种基因治疗，一些问题仍然没有答案，特别是当基因疗法在幼儿中进行时。我们在这里回顾了有关小儿眼科基因治疗各种方法的最新报道和有希望的研究。
