基因技术对遗传性和获得性视网膜变性进展的抑制作用。
Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques.
机构信息
Jonas Children's Vision Care and Bernard & Shirlee Brown Glaucoma Laboratory, New York, USA.
Department of Ophthalmology, Columbia University, New York, NY, USA.
出版信息
Mol Diagn Ther. 2019 Feb;23(1):113-120. doi: 10.1007/s40291-018-0377-1.
Abstract
Inherited retinal dystrophies cause progressive vision loss and are major contributors to blindness worldwide. Advances in gene therapy have brought molecular approaches into the realm of clinical trials for these incurable illnesses. Select phase I, II and III trials are complete and provide some promise in terms of functional outcomes and safety, although questions do remain over the durability of their effects and the prevalence of inflammatory reactions. This article reviews gene therapy as it can be applied to inherited retinal dystrophies, provides an update of results from recent clinical trials, and discusses the future prospects of gene therapy and genome surgery.
摘要
遗传性视网膜病变导致进行性视力丧失，是全球失明的主要原因。基因治疗的进步将分子方法引入了这些无法治愈疾病的临床试验领域。一些 I 期、II 期和 III 期临床试验已经完成，在功能结果和安全性方面提供了一些希望，尽管关于其效果的持久性和炎症反应的普遍性仍存在一些疑问。本文综述了基因治疗在遗传性视网膜病变中的应用，介绍了最近临床试验的结果，并讨论了基因治疗和基因组手术的未来前景。
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